Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy
Open accessAbstract
Fabry disease is a lysosomal storage disorder caused by the deficiency of α-galactosidase A. Enzyme deficiency results in a progressive decline in renal and cardiac function, leading to cardiomyopathy and end-stage renal disease. Current treatments available, including enzyme replacement therapies, have provided significant benefit to patients; however, unmet medical needs remain. mRNA therapy, with drug-like properties, has the unique ability to produce therapeutic proteins endogenously. Here we describe the sustained delivery of therapeutic human α-galactosidase protein in vivo via nanoparticle-formulated mRNA in mouse and non-human primate, with a demonstration of efficacy through clinically relevant biomarker reduction in a mouse Fabry disease model. Multi-component nanoparticles formulated with lipids and lipid-like materials were developed for the delivery of mRNA encoding human α-galactosidase protein. Upon delivery of human GLA mRNA to mice, serum GLA protein levels reached as high as ∼1,330-fold over normal physiological values.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The therapeutic potential of mRNA-based protein replacement therapies has been tested in other metabolic disorders in preclinical studies, including hereditary tyrosinaemia type I, acute intermittent porphyria, Fabry disease, Crigler-Najjar syndrome type 1, α1 antitrypsin deficiency, methylmalonic acidaemia/aciduria, arginase deficiency, citrin deficiency and glycogen storage disease type I.
“The therapeutic potential of mRNA-based protein replacement therapies has also been tested in other metabolic disorders in preclinical studies, including hereditary tyrosinaemia type I (HTI)142, acute intermittent porphyria222,223, Fabry disease224,225, Crigler–Najjar syndrome type 1 (ref.226), α1 antitrypsin deficiency227, methylmalonic acidaemia/aciduria228, arginase deficiency229, citrin defic”
From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [225]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms
Akinc, A., Querbes, W., De, S., Qin, J., Frank-Kamenetsky, M., Jayaprakash, K. N., Jayaraman, M., Rajeev, K. G., Cantley, W. L., Dorkin, J. R., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.85 · Cited by 1,261 · see citing works
The Many Pathways of RNA Degradation
Houseley, J. M., & Tollervey, D. W. (2009) · Cell · doi:10.1016/j.cell.2009.01.019 · Cited by 1,220 · see citing works
Pardi, N., Tuyishime, S., Muramatsu, H., Kariko, K., Mui, B. L., Tam, Y. K., Madden, T. D., Hope, M. J., & Weissman, D. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2015.08.007 · Cited by 1,014 · see citing works
Lipid-like materials for low-dose, in vivo gene silencing
Love, K. T., Mahon, K. P., Levins, C. G., Whitehead, K. A., Querbes, W., Dorkin, J. R., Qin, J., Cantley, W., Qin, L. L., Racie, T., et al. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0910603106 · Cited by 969 · see citing works
Kauffman, K. J., Dorkin, J. R., Yang, J. H., Heartlein, M. W., DeRosa, F., Mir, F. F., Fenton, O. S., & Anderson, D. G. (2015) · Nano Letters · doi:10.1021/acs.nanolett.5b02497 · Cited by 785 · see citing works
Self-citationExpression of therapeutic proteins after delivery of chemically modified mRNA in mice
Kormann, M. S. D., Hasenpusch, G., Aneja, M. K., Nica, G., Flemmer, A. W., Herber-Jonat, S., Huppmann, M., Mays, L. E., Illenyi, M., Schams, A., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1733 · Cited by 733 · see citing works
Zangi, L., Lui, K. O., von Gise, A., Ma, Q., Ebina, W., Ptaszek, L. M., Später, D., Xu, H., Tabebordbar, M., Gorbatov, R., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2682 · Cited by 646 · see citing works
Stability of Endogenous and Added RNA in Blood Specimens, Serum, and Plasma
Tsui, N. B. Y., Ng, E. K. O., & Lo, Y. D. (2002) · Clinical Chemistry · doi:10.1093/clinchem/48.10.1647 · Cited by 628 · see citing works
Intracellular targeting delivery of liposomal drugs to solid tumors based on EPR effects
Maruyama, K. (2010) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2010.09.003 · Cited by 603 · see citing works
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
Germain, D. P., Hughes, D. A., Nicholls, K., Bichet, D. G., Giugliani, R., Wilcox, W. R., Feliciani, C., Shankar, S. P., Ezgü, F. S., Amartino, H. M., et al. (2016) · New England Journal of Medicine · doi:10.1056/nejmoa1510198 · Cited by 555 · see citing works
Alberer, M., Gnad-Vogt, U., Hong, H. S., Mehr, K. T., Backert, L., Finak, G., Gottardo, R., Bica, M. A., Garofano, A., Koch, S. D., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)31665-3 · Cited by 530 · see citing works
Getting the glycosylation right: Implications for the biotechnology industry
Jenkins, N., Parekh, R. B., & James, D. C. (1996) · Nature Biotechnology · doi:10.1038/nbt0896-975 · Cited by 458 · see citing works
α-Galactosidase A deficient mice: A model of Fabry disease
Ohshima, T., Murray, G. J., Swaim, W. D., Longenecker, G., Quirk, J. M., CARDARELLI, C. O., Sugimoto, Y., Pastan, I. H., Gottesman, M. M., Brady, R. O., et al. (1997) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.94.6.2540 · Cited by 366 · see citing works
Pardi, N., Secreto, A. J., Shan, X., Debonera, F., Glover, J., Yi, Y., Muramatsu, H., Ni, H., Mui, B. L., Tam, Y. K., et al. (2017) · Nature Communications · doi:10.1038/ncomms14630 · Cited by 352 · see citing works
Schiffmann, R., Murray, G. J., Treco, D., Daniel, P., Sellos-Moura, M., Myers, M., Quirk, J. M., Zirzow, G. C., Borowski, M., Loveday, K. S., et al. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.1.365 · Cited by 341 · see citing works
Elimination of large tumors in mice by mRNA-encoded bispecific antibodies
Stadler, C. R., Bähr-Mahmud, H., Celik, L., Hebich, B., Roth, A. S., Roth, R. P., Karikó, K., Türeci, Ö., & Sahin, U. (2017) · Nature Medicine · doi:10.1038/nm.4356 · Cited by 290 · see citing works
Karikó, K., Muramatsu, H., Keller, J. M., & Weissman, D. (2012) · Molecular Therapy · doi:10.1038/mt.2012.7 · Cited by 282 · see citing works
Enzyme therapy for Fabry disease: Neutralizing antibodies toward agalsidase alpha and beta
Linthorst, G. E., Hollak, C. E. M., Donker‐Koopman, W. E., Strijland, A., & Aerts, J. M. F. G. (2004) · Kidney International · doi:10.1111/j.1523-1755.2004.00924.x · Cited by 280 · see citing works
Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis
Robinson, E., MacDonald, K. D., Slaughter, K., McKinney, M., Patel, S., Sun, C., & Sahay, G. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.05.014 · Cited by 272 · see citing works
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
An, D., Schneller, J. L., Frassetto, A., Liang, S., Zhu, X., Park, J.-S., Theisen, M., Hong, S.-J., Zhou, J., Rajendran, R., et al. (2017) · Cell Reports · doi:10.1016/j.celrep.2017.11.081 · Cited by 263 · see citing works
An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo
Li, B., Luo, X., Deng, B., Wang, J., McComb, D. W., Shi, Y., Gaensler, K. M. L., Tan, X., Dunn, A. L., Kerlin, B. A., et al. (2015) · Nano Letters · doi:10.1021/acs.nanolett.5b03528 · Cited by 261 · see citing works
Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery
Fenton, O. S., Kauffman, K. J., McClellan, R. L., Appel, E. A., Dorkin, J. R., Tibbitt, M. W., Heartlein, M. W., DeRosa, F., Langer, R., & Anderson, D. G. (2016) · Advanced Materials · doi:10.1002/adma.201505822 · Cited by 254 · see citing works
Self-citationRenal Pathology in Fabry Disease
Alroy, J., Sabnis, S. G., & Kopp, J. B. (2002) · Journal of the American Society of Nephrology · doi:10.1097/01.asn.0000016684.07368.75 · Cited by 244 · see citing works
Schiffmann, R., Hughes, D. A., Linthorst, G. E., Ortíz, A., Svarstad, E., Warnock, D. G., West, M. L., Wanner, C., Bichet, D. G., Christensen, E., et al. (2016) · Kidney International · doi:10.1016/j.kint.2016.10.004 · Cited by 196 · see citing works
Wang, Y.-H., Su, H.-H., Yang, Y., Hu, Y.-X., Zhang, L., Blancafort, P., & Huang, L. (2013) · Molecular Therapy · doi:10.1038/mt.2012.250 · Cited by 189 · see citing works
Systemic messenger RNA as an etiological treatment for acute intermittent porphyria
Jiang, L., Berraondo, P., Jericó, D., Guey, L. T., Sampedro, A., Frassetto, A., Benenato, K. E., Burke, K., Santamaría, E., Alegre, M., et al. (2018) · Nature Medicine · doi:10.1038/s41591-018-0199-z · Cited by 181 · see citing works
Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system
DeRosa, F., Guild, B., Karve, S., Smith, L., Love, K., Dorkin, J. R., Kauffman, K. J., Zhang, J., Yahalom, B., Anderson, D. G., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.46 · Cited by 159 · see citing works
Self-citationZhao, X. (2004) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2004.01.005 · Cited by 153 · see citing works
Efficient gene silencing in metastatic tumor by siRNA formulated in surface-modified nanoparticles
Li, S., Chono, S., & Huang, L. (2007) · Journal of Controlled Release · doi:10.1016/j.jconrel.2007.11.002 · Cited by 147 · see citing works
Nabhan, J. F., Wood, K. M., Rao, V. P., Morin, J., Bhamidipaty, S., LaBranche, T. P., Gooch, R. L., Bozal, F., Bulawa, C. E., & Guild, B. C. (2016) · Scientific Reports · doi:10.1038/srep20019 · Cited by 140 · see citing works
Engineered mRNA-expressed antibodies prevent respiratory syncytial virus infection
Tiwari, P. M., Vanover, D. A., Lindsay, K. E., Bawage, S. S., Kirschman, J. L., Bhosle, S. M., Lifland, A. W., Zurla, C., & Santangelo, P. J. (2018) · Nature Communications · doi:10.1038/s41467-018-06508-3 · Cited by 139 · see citing works
Plasma LysoGb3: A useful biomarker for the diagnosis and treatment of Fabry disease heterozygotes
Nowak, A., Mechtler, T. P., Desnick, R. J., & Kasper, D. C. (2016) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2016.10.006 · Cited by 136 · see citing works
Lipid-Based Nanoparticles in The Systemic Delivery of siRNA
Lin, Q., Chen, J., Zhang, Z., & Zheng, G. (2013) · Nanomedicine · doi:10.2217/nnm.13.192 · Cited by 133 · see citing works
Modified Foxp3 mRNA protects against asthma through an IL-10-dependent mechanism
Mays, L. E., Ammon-Treiber, S., Mothes, B., Alkhaled, M., Rottenberger, J., Müller-Hermelink, E. S., Grimm, M., Mezger, M., Beer-Hammer, S., von Stebut, E., et al. (2013) · Journal of Clinical Investigation · doi:10.1172/jci65351 · Cited by 113 · see citing works
Anderson-Fabry Disease and the Heart
O’Mahony, C., & Elliot, P. M. (2010) · Progress in Cardiovascular Diseases · doi:10.1016/j.pcad.2009.11.002 · Cited by 100 · see citing works
Chen, C., Lu, D., Yeh, Y., Shiau, & Chiang (2011) · International Journal of Nanomedicine · doi:10.2147/ijn.s24447 · Cited by 94 · see citing works
Innate and Adaptive Immune Response in Fabry Disease
Mauhin, W., Lidove, O., Masat, E., Mingozzi, F., Mariampillai, K., Ziza, J.-M., & Benvéniste, O. (2015) · JIMD Reports · doi:10.1007/8904_2014_371 · Cited by 85 · see citing works
Transcript-activated collagen matrix as sustained mRNA delivery system for bone regeneration
Badieyan, Z. S., Berezhanskyy, T., Utzinger, M., Aneja, M. K., Emrich, D., Erben, R. G., Schüler, C., Altpeter, P., Ferizi, M., Hasenpusch, G., et al. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.08.037 · Cited by 80 · see citing works
Recognition of mRNA cap structures by viral and cellular proteins
Fechter, P., & Brownlee, G. G. (2005) · Journal of General Virology · doi:10.1099/vir.0.80755-0 · Cited by 75 · see citing works
Roseman, D. S., Khan, T., Rajas, F., Jun, L. S., Asrani, K. H., Isaacs, C., Farelli, J. D., & Subramanian, R. R. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.01.006 · Cited by 72 · see citing works
Consequences of a global enzyme shortage of agalsidase beta in adult Dutch Fabry patients
Smid, B. E., Rombach, S. M., Aerts, J. M. F. G., Kuiper, S., Mirzaian, M., Overkleeft, H. S., Poorthuis, B. J. H. M., Hollak, C. E. M., Groener, J. E. M., & Linthorst, G. E. (2011) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-6-69 · Cited by 71 · see citing works
McCaskill, J. L., Singhania, R., Burgess, M. L., Allavena, R., Wu, S. Y., Blumenthal, A., & McMillan, N. A. J. (2013) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2013.22 · Cited by 70 · see citing works
Schrom, E., Huber, M., Aneja, M., Dohmen, C., Emrich, D., Geiger, J., Hasenpusch, G., Herrmann-Janson, A., Kretzschmann, V., Mykhailyk, O., et al. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.04.006 · Cited by 69 · see citing works
Bangel‐Ruland, N., Tomczak, K., Fernández, E. F., Leier, G., Leciejewski, B., Rudolph, C., Rosenecker, J., & Weber, W. (2013) · The Journal of Gene Medicine · doi:10.1002/jgm.2748 · Cited by 67 · see citing works
SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency
Connolly, B., Isaacs, C., Cheng, L., Asrani, K. H., & Subramanian, R. R. (2018) · Journal of Nucleic Acids · doi:10.1155/2018/8247935 · Cited by 67 · see citing works
Arginase I mRNA therapy – a novel approach to rescue arginase 1 enzyme deficiency
Asrani, K. H., Cheng, L., Cheng, C. J., & Subramanian, R. R. (2018) · RNA Biology · doi:10.1080/15476286.2018.1475178 · Cited by 58 · see citing works
Novel Gb3 Isoforms Detected in Urine of Fabry Disease Patients: A Metabolomic Study
Auray‐Blais, C., & Boutin, M. (2012) · Current Medicinal Chemistry · doi:10.2174/092986712800784739 · Cited by 50 · see citing works
Young-Gqamana, B., Brignol, N., Chang, H., Khanna, R., Soska, R., Fuller, M., Sitaraman, S., Germain, D. P., Giugliani, R., Hughes, D. A., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0057631 · Cited by 49 · see citing works
Fabry Disease – Current Treatment and New Drug Development
Motabar, O., Sidransky, E., Goldin, E., & Zheng, W. (2010) · Current Chemical Genomics · doi:10.2174/1875397301004010050 · Cited by 48 · see citing works
Linthorst, G. E., Germain, D. P., Hollak, C. E. M., Hughes, D. A., Rolfs, A., Wanner, C., & Mehta, A. B. (2010) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2010.11.155 · Cited by 47 · see citing works
Renal manifestations in Fabry disease and therapeutic options
Torrá, R. (2008) · Kidney International · doi:10.1038/ki.2008.522 · Cited by 46 · see citing works
Xu, Y., Ou, M., Keough, E., Roberts, J., Koeplinger, K., Lyman, M., Fauty, S. E., Carlini, E., Stern, M., Zhang, R., et al. (2014) · Molecular Pharmaceutics · doi:10.1021/mp400584h · Cited by 38 · see citing works
Long Term Treatment with Enzyme Replacement Therapy in Patients with Fabry Disease
Oder, D., Nordbeck, P., & Wanner, C. (2016) · The Nephron journals/Nephron journals · doi:10.1159/000448968 · Cited by 37 · see citing works
Keating, G. M., & Simpson, D. (2007) · Drugs · doi:10.2165/00003495-200767030-00007 · Cited by 30 · see citing works
Severe Infusion Reactions to Fabry Enzyme Replacement Therapy: Rechallenge After Tracheostomy
Nicholls, K., Bleasel, K. F., & Becker, G. J. (2011) · JIMD Reports · doi:10.1007/8904_2011_106 · Cited by 27 · see citing works
Biomarkers and Imaging Findings of Anderson–Fabry Disease—What We Know Now
Beirão, I. B., Cabrita, A., Torres, M., da Silva, F. A. D. A., Aguiar, P., Laranjeira, F., & Gomes, A. M. (2017) · Diseases · doi:10.3390/diseases5020015 · Cited by 27 · see citing works
Fabry disease and its cardiac involvement
Kubo, T. (2017) · Journal of General and Family Medicine · doi:10.1002/jgf2.76 · Cited by 25 · see citing works
Tendon healing induced by chemically modified mRNAs
Groth, K., Berezhanskyy, T., Aneja, M., Geiger, J. P., Schweizer, M., Maucksch, L., Pasewald, T., Brill, T., Tigani, B., Weber, E., et al. (2017) · European Cells and Materials · doi:10.22203/ecm.v033a22 · Cited by 25 · see citing works
Bioprocess factors affecting glycoprotein oligosaccharide structure.
Goochee, C. F. (1992) · PubMed · Cited by 22 · see citing works
Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies
Antony, J. S., Dewerth, A., Haque, A., Handgretinger, R., & Kormann, M. (2015) · Molecular and Cellular Pediatrics · doi:10.1186/s40348-015-0022-6 · Cited by 18 · see citing works
Effect of Reduced Agalsidase Beta Dosage in Fabry Patients: The Australian Experience
Centres, A. S. F. D. T., Ghali, J. R., Nicholls, K., Denaro, C. P., Sillence, D. O., Chapman, I. M., Goldblatt, J., Thomas, M., & Fletcher, J. M. (2011) · JIMD Reports · doi:10.1007/8904_2011_44 · Cited by 15 · see citing works
Ishii, S., Kase, R., Sakuraba, H., Taya, C., Yonekawa, H., Okumiya, T., Matsuda, Y., Mannen, K., Takeshita, M., & Suzuki, Y. (1998) · Glycoconjugate Journal · doi:10.1023/a:1006915926732 · Cited by 14 · see citing works
Agalsidase alfa (Replagal) in the treatment of Anderson-Fabry disease.
Pastores, G. M. (2007) · PubMed · Cited by 10 · see citing works
Fabry disease: is there a role for enzyme replacement therapy?
Mehta, A. B. (2013) · Journal of Internal Medicine · doi:10.1111/joim.12089 · Cited by 6 · see citing works
Agalsidase alfa and agalsidase beta in the treatment of Fabry disease: does the dose really matter?
Pisani, A., Riccio, E., & Sabbatini, M. (2014) · Genetics in Medicine · doi:10.1038/gim.2014.79 · Cited by 6 · see citing works
Enzyme Replacement Therapy for Fabry Disease
Sánchez-Niño, M. D., & Ortíz, A. (2016) · Journal of Inborn Errors of Metabolism and Screening · doi:10.1177/2326409816679428 · Cited by 3 · see citing works
Agalsidase alfa for the treatment of fabry disease: a closer look
Lipinski, S. E., Komlódi-Pásztor, E., & Göker-Alpan, Ö. (2014) · Clinical Investigation · doi:10.4155/cli.14.35 · Cited by 1 · see citing works
Flagged first, then by citations
1 of the 68 references couldn’t be matched to a known paper.
Cited by
Sorted by citations
Lipid nanoparticles for mRNA delivery
Hou, X., Zaks, T., Langer, R., & Dong, Y. (2021) · Nature Reviews Materials · doi:10.1038/s41578-021-00358-0 · Cited by 4,076 · see citing works
mRNA-based therapeutics: powerful and versatile tools to combat diseases
Qin, S., Tang, X., Chen, Y., Chen, K., Fan, N., Xiao, W., Zheng, Q., Li, G., Teng, Y., Wu, M., et al. (2022) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-022-01007-w · Cited by 801 · see citing works
Lipids and Lipid Derivatives for RNA Delivery
Zhang, Y., Sun, C., Wang, C., Jankovic, K. E., & Dong, Y. (2021) · Chemical Reviews · doi:10.1021/acs.chemrev.1c00244 · Cited by 638 · see citing works
Lipid nanoparticles for nucleic acid delivery: Current perspectives
Samaridou, E., Heyes, J. A., & Lutwyche, P. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.06.002 · Cited by 593 · see citing works
Nanomaterial Delivery Systems for mRNA Vaccines
Buschmann, M. D., Carrasco, M. J., Alishetty, S., Paige, M., Alameh, M. G., & Weissman, D. (2021) · Vaccines · doi:10.3390/vaccines9010065 · Cited by 582 · see citing works
The promise of mRNA vaccines: a biotech and industrial perspective
Jackson, N. A. C., Kester, K. E., Casimiro, D., Gurunathan, S., & DeRosa, F. (2020) · npj Vaccines · doi:10.1038/s41541-020-0159-8 · Cited by 498 · see citing works
“[34] [35] [36] There have been numerous examples of successful delivery of mRNA by using LNPs for therapeutic [37] [38] [39] [40] as well as vaccine applications.”
BackgroundSelf-citationCardiac Involvement in Fabry Disease
Pieroni, M., Moon, J., Arbustini, E., Villa, R. B., Camporeale, A., Vujkovac, A. C., Elliot, P. M., Hagège, A. A., Kuusisto, J., Linhart, A., et al. (2021) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2020.12.024 · Cited by 347 · see citing works
Zhang, R., El-Mayta, R. D., Murdoch, T. J., Warzecha, C. C., Billingsley, M. M., Shepherd, S. J., Gong, N., Wang, L., Wilson, J. M., Lee, D., et al. (2020) · Biomaterials Science · doi:10.1039/d0bm01609h · Cited by 261 · see citing works
Jeong, M., Lee, Y., Park, J., Jung, H., & Lee, H. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.114990 · Cited by 227 · see citing works
Progress and prospects of mRNA-based drugs in pre-clinical and clinical applications
Shi, Y., Shi, M., Wáng, Y., & You, J. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-02002-z · Cited by 231 · see citing works
Developments in the treatment of Fabry disease
van der Veen, S. J., Hollak, C. E. M., Kuilenburg, A. B. P., & Langeveld, M. (2020) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.12228 · Cited by 153 · see citing works
mRNA in the Context of Protein Replacement Therapy
Vavilis, T., Stamoula, Ε., Ainatzoglou, A., Sachinidis, A., Lamprinou, M., Dardalas, I., & Vizirianakis, I. S. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15010166 · Cited by 143 · see citing works
Nanomedicine-Based Approaches for mRNA Delivery
Uchida, S., Perche, F., Pichon, C., & Cabral, H. (2020) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.0c00618 · Cited by 139 · see citing works
mRNA nanomedicine: Design and recent applications
Kubiatowicz, L. J., Mohapatra, A., Krishnan, N., Fang, R. H., & Zhang, L. (2022) · Exploration · doi:10.1002/exp.20210217 · Cited by 130 · see citing works
Development of multivalent mRNA vaccine candidates for seasonal or pandemic influenza
Chivukula, S., Plitnik, T., Tibbitts, T., Karve, S., Dias, A., Zhang, D., Goldman, R., Gopani, H., Khanmohammed, A., Sarode, A., et al. (2021) · npj Vaccines · doi:10.1038/s41541-021-00420-6 · Cited by 117 · see citing works
Self-citationProgress in the understanding and treatment of Fabry disease
Miller, J. J., Kanack, A. J., & Dahms, N. M. (2019) · Biochimica et Biophysica Acta (BBA) - General Subjects · doi:10.1016/j.bbagen.2019.129437 · Cited by 111 · see citing works
Non-immunotherapy application of LNP-mRNA: Maximizing efficacy and safety
Vlatkovic, I. (2021) · Biomedicines · doi:10.3390/biomedicines9050530 · Cited by 112 · see citing works
Current and Investigational Therapeutics for Fabry Disease
Felis, A., Whitlow, M., Kraus, A., Warnock, D. G., & Wallace, E. (2020) · Kidney International Reports · doi:10.1016/j.ekir.2019.11.013 · Cited by 100 · see citing works
Haghighi, E., Abolmaali, S. S., Dehshahri, A., Shaegh, S. A. M., Azarpira, N., & Tamaddon, A. M. (2024) · Journal of Nanobiotechnology · doi:10.1186/s12951-024-02972-w · Cited by 110 · see citing works
Fabry Disease Therapy: State-of-the-Art and Current Challenges
Azevedo, O. M., Gago, M. F., Miltenberger-Miltényi, G., Sousa, N., & Cunha, D. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms22010206 · Cited by 91 · see citing works
Frameworks for transformational breakthroughs in RNA-based medicines
Androsavich, J. R. (2024) · Nature Reviews Drug Discovery · doi:10.1038/s41573-024-00943-2 · Cited by 93 · see citing works
Ma, Y., Cai, F., Li, Y., Chen, J., Han, F., & Lin, W. (2020) · Bioactive Materials · doi:10.1016/j.bioactmat.2020.05.002 · Cited by 89 · see citing works
Immunogenicity and efficacy of mRNA COVID-19 vaccine MRT5500 in preclinical animal models
Kalnin, K. V., Plitnik, T., Kishko, M., Zhang, J., Zhang, D., Beauvais, A., Anosova, N. G., Tibbitts, T., DiNapoli, J., Ulinski, G., et al. (2021) · npj Vaccines · doi:10.1038/s41541-021-00324-5 · Cited by 82 · see citing works
Self-citationRNA delivery biomaterials for the treatment of genetic and rare diseases
Zhao, W., Hou, X., Vick, O. G., & Dong, Y. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119291 · Cited by 82 · see citing works
Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics
Semple, S. C., Leone, R. D., Barbosa, C., Tam, Y. K., & Lin, P. J. C. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14020398 · Cited by 80 · see citing works
Lipid Nanoparticles: A Novel Gene Delivery Technique for Clinical Application
Mashima, R., & Takada, S. (2022) · Current Issues in Molecular Biology · doi:10.3390/cimb44100341 · Cited by 68 · see citing works
Ionizable Lipid Nanoparticles for mRNA Delivery
Tang, X., Zhang, Y., & Han, X. (2023) · Advanced NanoBiomed Research · doi:10.1002/anbr.202300006 · Cited by 71 · see citing works
mRNA therapies: Pioneering a new era in rare genetic disease treatment
Shen, G., Liu, J., Yang, H., Xie, N., & Yang, Y. (2024) · Journal of Controlled Release · doi:10.1016/j.jconrel.2024.03.056 · Cited by 61 · see citing works
Inhalable dry powder product (DPP) of mRNA lipid nanoparticles (LNPs) for pulmonary delivery
Sarode, A., Patel, P., Vargas‐Montoya, N., Allawzi, A. M., Zhilin-Roth, A., Karmakar, S., Boeglin, L., Deng, H., Karve, S., & DeRosa, F. (2023) · Drug Delivery and Translational Research · doi:10.1007/s13346-023-01402-y · Cited by 64 · see citing works
Self-citationGene Therapy for Methylmalonic Acidemia: Past, Present, and Future
Chandler, R. J., & Venditti, C. P. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.113 · Cited by 54 · see citing works
Targeting strategies for mRNA delivery
Meyer, R. A., Neshat, S. Y., Green, J. J., Santos, J. L., & Tuesca, A. D. (2022) · Materials Today Advances · doi:10.1016/j.mtadv.2022.100240 · Cited by 53 · see citing works
Li, X., Li, J., Wei, J., Du, W., Su, C., Shen, X., Zhao, A., & Xu, M. (2025) · MedComm · doi:10.1002/mco2.70414 · Cited by 56 · see citing works
High‐Precision Synthesis of RNA‐Loaded Lipid Nanoparticles for Biomedical Applications
Seo, H., Jeon, L., Kwon, J., & Lee, H. (2023) · Advanced Healthcare Materials · doi:10.1002/adhm.202203033 · Cited by 51 · see citing works
Cellular Trafficking of Nanotechnology‐Mediated mRNA Delivery
Huang, P., Deng, H., Wang, C., Zhou, Y., & Chen, X. S. (2023) · Advanced Materials · doi:10.1002/adma.202307822 · Cited by 49 · see citing works
Fabry Disease: Current and Novel Therapeutic Strategies. A Narrative Review
Palaiodimou, L., Kokotis, P., Zompola, C., Papagiannopoulou, G., Bakola, E., Papadopoulou, Μ., Zouvelou, V., Petras, D. I., Vlachopoulos, C., & Tsivgoulis, G. K. (2022) · Current Neuropharmacology · doi:10.2174/1570159x20666220601124117 · Cited by 47 · see citing works
Nanoscale delivery platforms for RNA therapeutics: Challenges and the current state of the art
Rhym, L. H., & Anderson, D. G. (2022) · Med · doi:10.1016/j.medj.2022.02.001 · Cited by 45 · see citing works
Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing
Domm, J. M., Wootton, S. K., Medin, J. A., & West, M. L. (2021) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2021.07.006 · Cited by 44 · see citing works
Fabry disease: Mechanism and therapeutics strategies
Li, X., Ren, X., Zhang, Y., Ding, L., Huo, M., & Li, Q. (2022) · Frontiers in Pharmacology · doi:10.3389/fphar.2022.1025740 · Cited by 43 · see citing works
Deyhimfar, R., Izady, M., Shoghi, M., Kazazi, M. H., Ghazvini, Z. F., Nazari, H., Fekrirad, Z., & Arefian, E. (2024) · Heliyon · doi:10.1016/j.heliyon.2024.e26971 · Cited by 40 · see citing works
Treatment of Fabry Disease: Established and Emerging Therapies
Umer, M., & Kalra, D. K. (2023) · Pharmaceuticals · doi:10.3390/ph16020320 · Cited by 38 · see citing works
Averbuch, T., White, J. A., & Fine, N. M. (2023) · Frontiers in Cardiovascular Medicine · doi:10.3389/fcvm.2023.1152568 · Cited by 37 · see citing works
mRNA and gene editing: Late breaking therapies in liver diseases
Zabaleta, N., Torella, L., Weber, N. D., & González‐Aseguinolaza, G. (2022) · Hepatology · doi:10.1002/hep.32441 · Cited by 36 · see citing works
Therapeutic advances in Fabry disease: The future awaits
Kant, S., & Atta, M. G. (2020) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2020.110779 · Cited by 38 · see citing works
mRNA-Enhanced Cell Therapy and Cardiovascular Regeneration
Chanda, P. K., Sukhovershin, R. A., & Cooke, J. P. (2021) · Cells · doi:10.3390/cells10010187 · Cited by 30 · see citing works
mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5
Hauser, S., Poenisch, M., Schelling, Y., Höflinger, P., Schuster, S., Teegler, A., Betten, R., Gustafsson, J.-Å., Hübener‐Schmid, J., Schlake, T., et al. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.10.011 · Cited by 28 · see citing works
An, J. H., Hong, S.-E., Yu, S., Kang, J., Park, C. G., Lee, H. Y., Lee, S. K., Lee, D. C., Park, H., Hwang, W.-M., et al. (2022) · Journal of Nanobiotechnology · doi:10.1186/s12951-022-01318-8 · Cited by 25 · see citing works
Goldman, R., Murthy, N. T. V., Trent, N., Balakrishnan, A., Chivukula, S. V., Danz, H., Tibbitts, T. J., Dias, A., Vargas, J., Cooper, D. L., et al. (2023) · Biomaterials · doi:10.1016/j.biomaterials.2023.122243 · Cited by 26 · see citing works
Self-citationα-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice
Rodríguez-Castejón, J., Alarcia-Lacalle, A., Gómez-Aguado, I., Vicente-Pascual, M., Solinís, M. Á., del Pozo‐Rodríguez, A., & Rodríguez‐Gascón, A. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13060771 · Cited by 24 · see citing works
Nonclinical safety evaluation of a novel ionizable lipid for mRNA delivery
Broudic, K., Amberg, A., Schaefer, M. K., Spirkl, H.-P., Bernard, M., & Désert, P. (2022) · Toxicology and Applied Pharmacology · doi:10.1016/j.taap.2022.116143 · Cited by 25 · see citing works
RNA-based therapies in liver metabolic diseases
Fontanellas, A., Berraondo, P., Urigo, F., Jericó, D., Martini, P. G. V., Pastor, F., & Avila, M. A. (2025) · Gut · doi:10.1136/gutjnl-2023-331742 · Cited by 23 · see citing works
Kim, I. G., Jung, W. H., You, G., Lee, H., Shin, Y., Lim, S. W., Chung, B. H., & Mok, H. (2023) · Macromolecular Bioscience · doi:10.1002/mabi.202200423 · Cited by 22 · see citing works
Progress and Challenges in the Treatment of Fabry Disease
Lenders, M., Menke, E. R., & Brand, E. (2025) · BioDrugs · doi:10.1007/s40259-025-00723-3 · Cited by 20 · see citing works
The Potential of Nanomedicine to Unlock the Limitless Applications of mRNA
Taina-González, L., & de la Fuente, M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14020460 · Cited by 21 · see citing works
Unleashing the potential of mRNA therapeutics for inherited neurological diseases
Monfrini, E., Baso, G., Ronchi, D., Meneri, M., Gagliardi, D., Quetti, L., Verde, F., Ticozzi, N., Ratti, A., Fonzo, A. D., et al. (2024) · Brain · doi:10.1093/brain/awae135 · Cited by 19 · see citing works
Immunogenicity of novel mRNA COVID-19 vaccine MRT5500 in mice and non-human primates
Kalnin, K. V., Plitnik, T., Kishko, M., Zhang, J., Zhang, D., Beauvais, A., Anosova, N. G., Tibbitts, T., DiNapoli, J. M., Huang, P.-W. D., et al. (2020) · doi:10.1101/2020.10.14.337535 · Cited by 18 · see citing works
Self-citationFabry Cardiomyopathy: Current Treatment and Future Options
Vardarli, I., Weber, M., Rischpler, C., Führer, D., Herrmann, K., & Weidemann, F. (2021) · Journal of Clinical Medicine · doi:10.3390/jcm10143026 · Cited by 18 · see citing works
Fabry disease: a rare disorder calling for personalized medicine
Lerario, S., Monti, L., Ambrosetti, I., Luglio, A., Pietra, A., Aiello, V., Montanari, F., Bellasi, A., Zaza, G., Galante, A., et al. (2024) · International Urology and Nephrology · doi:10.1007/s11255-024-04042-4 · Cited by 18 · see citing works
Emerging mRNA Technology for Liver Disease Therapy
Wang, J., Fang, Y., Luo, Z., Wang, J., & Zhao, Y. (2024) · ACS Nano · doi:10.1021/acsnano.4c02987 · Cited by 19 · see citing works
Models to study basic and applied aspects of lysosomal storage disorders
Gaudioso, Á., Silva, T. P., & Ledesma, M. D. (2022) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114532 · Cited by 18 · see citing works
Anderson–Fabry Disease: From Endothelial Dysfunction to Emerging Therapies
Stamerra, C. A., Pinto, R. D., Giosia, P. D., Ferri, C., & Sahebkar, A. (2021) · Advances in Pharmacological and Pharmaceutical Sciences · doi:10.1155/2021/5548445 · Cited by 16 · see citing works
Current and experimental therapeutics for Fabry disease
Castelli, V., Stamerra, C. A., d’Angelo, M., Cimini, A., & Ferri, C. (2021) · Clinical Genetics · doi:10.1111/cge.13999 · Cited by 16 · see citing works
Muntean, C. V., Stârcea, I. M., Stoica, C. I., & Bănescu, C. V. (2022) · Frontiers in Pediatrics · doi:10.3389/fped.2022.908657 · Cited by 15 · see citing works
mRNA biotherapeutics landscape for rare genetic disorders
Iyer, V. R., Praveen Reddy., P., Kaduskar, B., Moharir, S. C., & Mishra, R. K. (2024) · Journal of Biosciences · doi:10.1007/s12038-023-00415-6 · Cited by 16 · see citing works
Immunology of RNA-based vaccines: The critical interplay between inflammation and expression
Tregoning, J. S., Wang, Z., Sridhar, S., Shattock, R. J., & DeRosa, F. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.09.011 · Cited by 19 · see citing works
Self-citationDanz, H., Dauner, A. L., Sharma, S., Babiceanu, M., Clark, N., Ontiveros, R. J., Amezquita, R. A., Zhilin-Roth, A., Reyes, E., Khoo, C., et al. (2025) · npj Vaccines · doi:10.1038/s41541-025-01263-1 · Cited by 14 · see citing works
Self-citationMonda, E., Falco, L., Palmiero, G., Rubino, M., Perna, A., Diana, G., Verrillo, F., Dongiglio, F., Cirillo, A., Fusco, A., et al. (2023) · Cardiac failure review · doi:10.15420/cfr.2023.06 · Cited by 14 · see citing works
Tahir, D., Geolier, V., Bruant, H., Flèche‐Matéos, A. L., Mallet, A., Varloud, M., Civat, C., Girerd-Chambaz, Y., Montano, S., Pion, C., et al. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102514 · Cited by 15 · see citing works
Advances in Development of mRNA-Based Therapeutics
Huang, L., Zhang, L., Li, W., Li, S., Wen, J., Li, H., & Liu, Z. (2020) · Current topics in microbiology and immunology · doi:10.1007/82_2020_222 · Cited by 14 · see citing works
Delaleu, N., Marti, H., Strauss, P., Sekulic, M., Osman, T., Tøndel, C., Skrunes, R., Leh, S. M., Svarstad, E., Nowak, A., et al. (2023) · Kidney International · doi:10.1016/j.kint.2023.06.029 · Cited by 12 · see citing works
Targeted ADAMTS-13 replacement therapy for thrombotic thrombocytopenic purpura
Moroniti, J. J., Vrbensky, J. R., Nazy, I., & Arnold, D. M. (2023) · Journal of Thrombosis and Haemostasis · doi:10.1016/j.jtha.2023.11.030 · Cited by 12 · see citing works
Galactomannan-Decorated Lipidic Nanocarrier for Gene Supplementation Therapy in Fabry Disease
Rodríguez-Castejón, J., Gómez-Aguado, I., Beraza-Millor, M., Solinís, M. Á., del Pozo‐Rodríguez, A., & Rodríguez‐Gascón, A. (2022) · Nanomaterials · doi:10.3390/nano12142339 · Cited by 11 · see citing works
Oder, D., Müntze, J., & Nordbeck, P. (2021) · Cardiovascular Diagnosis and Therapy · doi:10.21037/cdt-20-743 · Cited by 10 · see citing works
Messenger RNA as a personalized therapy: The moment of truth for rare metabolic diseases
Córdoba, K. M., Jericó, D., Sampedro, A., Jiang, L., Iraburu, M. J., Martini, P. G. V., Berraondo, P., Avila, M. A., & Fontanellas, A. (2022) · International review of cytology · doi:10.1016/bs.ircmb.2022.03.005 · Cited by 11 · see citing works
Deng, Y., Atyeo, C., Yuan, D., Chicz, T., Tibbitts, T., Gorman, M., Taylor, S., Lecouturier, V., Lauffenburger, D., Chicz, R., et al. (2023) · Cell Reports · doi:10.1016/j.celrep.2023.113292 · Cited by 9 · see citing works
Carella, M. C., Forleo, C., Caretto, P., Naccarati, M. L., Dentamaro, I., Dicorato, M. M., Basile, P., Carulli, E., Latorre, M. D., Baggiano, A., et al. (2024) · Journal of Clinical Medicine · doi:10.3390/jcm13237195 · Cited by 10 · see citing works
Kalnin, K. V., Plitnik, T., Kishko, M., Huang, D., Raillard, A., Piolat, J., Anosova, N. G., Tibbitts, T. J., DiNapoli, J. M., Karve, S., et al. (2022) · Vaccine · doi:10.1016/j.vaccine.2022.01.021 · Cited by 10 · see citing works
Self-citationRodríguez-Castejón, J., Beraza-Millor, M., Solinís, M. Á., Rodríguez‐Gascón, A., & del Pozo‐Rodríguez, A. (2024) · Drug Delivery and Translational Research · doi:10.1007/s13346-024-01583-0 · Cited by 8 · see citing works
Cellular functions and biomedical applications of circular RNAs
Zhang, Z., & Wang, Z. (2024) · Acta Biochimica et Biophysica Sinica · doi:10.3724/abbs.2024241 · Cited by 9 · see citing works
Human in vitro models for Fabry disease: new paths for unravelling disease mechanisms and therapies
Borisch, C., Thum, T., Bär, C., & Hoepfner, J. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05756-w · Cited by 7 · see citing works
Future prospects in mRNA vaccine development
Mbatha, L. S., Akinyelu, J., Maiyo, F., & Kudanga, T. (2023) · Biomedical Materials · doi:10.1088/1748-605x/aceceb · Cited by 6 · see citing works
Hypertrophic Cardiomyopathy versus Storage Diseases with Myocardial Involvement
Burban, A., Pucyło, S., Sikora, A., Opolski, G., Grabowski, M., & Kołodzińska, A. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms241713239 · Cited by 5 · see citing works
Sarode, A., Ortiz, C., Derstepanian, T., Vargas‐Montoya, N., Patel, P., Khadse, N., Manna, S., Landis, R. F., Skaleski, J., Boeglin, L., et al. (2025) · Advanced Materials · doi:10.1002/adma.202517893 · Cited by 4 · see citing works
Self-citationCMC and regulatory aspects of oligonucleotide therapeutics
Rupp, T. M., & Cramer, H. (2022) · Elsevier eBooks · doi:10.1016/b978-0-12-821595-1.00012-9 · Cited by 4 · see citing works
Current and Emerging Therapies for Lysosomal Storage Disorders
Lastoria, D. A. A., Keynes, S., & Hughes, D. A. (2025) · Drugs · doi:10.1007/s40265-025-02145-5 · Cited by 4 · see citing works
Targeted Identification of Protein Interactions in Eukaryotic mRNA Translation
Link, A. J., Niu, X., Weaver, C. M., Jennings, J. L., Duncan, D. T., McAfee, K. J., Sammons, M. A., Gerbasi, V. R., Farley, A. R., Fleischer, T. C., et al. (2020) · PROTEOMICS · doi:10.1002/pmic.201900177 · Cited by 4 · see citing works
Cao, J., Markel, A., Hanahoe, E., Ketova, T., Mihai, C., Zalinger, Z., Marquardt, D., Amato, N. J., Cheng, Y. M., Reid, D. W., et al. (2023) · Amino Acids · doi:10.1007/s00726-023-03263-8 · Cited by 4 · see citing works
Alfarano, M., Ciccarelli, F., Marchionni, G., Ballatore, F., Costantino, J., Lattanzio, A., Pecci, G., Stavagna, S., Iannelli, L., Galardo, G., et al. (2025) · Journal of Clinical Medicine · doi:10.3390/jcm14124228 · Cited by 3 · see citing works
Zhang, M., & Wang, C. (2025) · Frontiers in Medicine · doi:10.3389/fmed.2025.1662867 · Cited by 3 · see citing works
Pioneering next-generation mRNA therapeutics through molecular engineering and delivery optimization
Qi, L., Li, Z., Feng, Z., Liu, J., & Chen, X. S. (2025) · Materials Today · doi:10.1016/j.mattod.2025.08.023 · Cited by 3 · see citing works
Biswas, G., Madhu, N. R., Sarkar, B., Paul, S., Erfani, H., & Alam, Q. (2024) · Springer eBooks · doi:10.1007/978-981-99-9323-9_7 · Cited by 2 · see citing works
The use of nanocarriers in treating Batten disease: A systematic review
Henke, L., Ghorbani, A., & Mole, S. (2024) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2024.125094 · Cited by 2 · see citing works
Pan, M., Cao, W., Zhai, J., Zheng, C., Xu, Y., & Zhang, P. (2025) · International Journal of Biological Macromolecules · doi:10.1016/j.ijbiomac.2025.143134 · Cited by 2 · see citing works
Gene Therapy in Anderson-Fabry Disease. State of the Art and Future Perspectives
Spiniello, G., Verrillo, F., Ricciolino, R., Prozzo, D., Tuccillo, A., Caiazza, M., & Rubino, M. (2020) · Cardiogenetics · doi:10.4081/cardiogenetics.2020.9075 · Cited by 2 · see citing works
Li, L., Wang, M., Ye, X., Liu, Z., Duan, Y., Chen, X., Ouyang, Y., Wu, Q., Sun, M., & Xie, T. (2026) · MedComm · doi:10.1002/mco2.70700 · Cited by 1 · see citing works
Genetic basis of cardiomyopathies
Thum, T., Heymans, S. R. B., & Backs, J. (2023) · doi:10.1093/med/9780198891628.003.0006 · Cited by 1 · see citing works
Development of DDS for mRNA therapeutics: Polyplex nanomicelle
Itaka, K. (2020) · Drug Delivery System · doi:10.2745/dds.35.27
Advanced drug delivery systems involving lysosomal storage disorders for Fabry disease
Chandran, C. S., Raj, A., Swathy, K. K., Sajayan, K., & Kappally, S. (2022) · Elsevier eBooks · doi:10.1016/b978-0-323-99616-7.00006-2
mRNA Vaccine for Infectious Disease Prevention and Cancer Immunotherapy
Dong, J. (2023) · Theoretical and Natural Science · doi:10.54254/2753-8818/3/20220236
Nierenbeteiligung bei Systemerkrankungen – Morbus Fabry
Lenders, M., & Brand, E. (2023) · Springer Reference Medizin · doi:10.1007/978-3-642-54676-1_66-2
- Authors
- DeRosa, F., Smith, L., Shen, Y., Huang, Y., Pan, J., Xie, H., Yahalom, B., & Heartlein, M. W.
- Affiliations
- Translate Bio (United States) (US)
- Shire (United States) (US)
- Corresponding author
- Heartlein, Michael W.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2019-04
- Volume
- 27
- Issue
- 4
- Pages
- 878-889
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC6453518
- OpenAlex
- W2922121037
- Cited by
- 111
- References
- 68
- Citations by year
- 2026: 7 · 2025: 14 · 2024: 18 · 2023: 21 · 2022: 19 · 2021: 14 · 2020: 13 · 2019: 5
- FWCI
- 8.74
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Funders
- Shire
- Topics
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Keywords
- Fabry disease, alpha-galactosidase A, mRNA therapeutics, sustained protein delivery, enzyme replacement therapy, lysosomal storage disease, nonhuman primate model
- MeSH terms
- alpha-Galactosidase, Fabry Disease, Animals, Callithrix, Disease Models, Animal, Drug Compounding, Female, Humans, Kidney, Lipids, Liver, Male, RNA, Messenger, Drug Delivery Systems, Treatment Outcome, Mice, Knockout, Mice, Nanoparticles, Gene Knockout Techniques, Enzyme Replacement Therapy
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
