Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

Coding of facial expressions of pain in the laboratory mouse

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A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

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Recommendations for the Diagnosis and Treatment of the Acute Porphyrias

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High Throughput Microplate Respiratory Measurements Using Minimal Quantities Of Isolated Mitochondria

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Porphyria

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Nutritional Regulation of Hepatic Heme Biosynthesis and Porphyria through PGC-1α

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Acute Porphyrias in the USA: Features of 108 Subjects from Porphyrias Consortium

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Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

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Givosiran: a targeted treatment for acute intermittent porphyria

Dickey, A. K., & Leaf, R. K. (2024) · Hematology · doi:10.1182/hematology.2024000663 · Cited by 18

Messenger RNA therapy as an option for treating metabolic disorders

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mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

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Treatment of Anderson-Fabry Disease

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Porphyrias in the Age of Targeted Therapies

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Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria

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Liver-directed gene-based therapies for inborn errors of metabolism

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Recent Insights into the Pathogenesis of Acute Porphyria Attacks and Increasing Hepatic PBGD as an Etiological Treatment

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Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model

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mRNA biotherapeutics landscape for rare genetic disorders

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Modified mRNA Treatment Restores Cardiac Function in Desmocollin-2–Deficient Mouse Models of Arrhythmogenic Right Ventricular Cardiomyopathy

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A Pharmacological Chaperone Therapy for Acute Intermittent Porphyria

Bustad, H. J., Toska, K., Schmitt, C., Vorland, M., Skjærven, L., Kallio, J. P., Simonin, S., Lettéron, P., Underhaug, J., Sandberg, S., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.11.010 · Cited by 15

Advances in Development of mRNA-Based Therapeutics

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The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism

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Models for human porphyrias: Have animals in the wild been overlooked?

Neves, A. C. D. O., & Galván, I. (2020) · BioEssays · doi:10.1002/bies.202000155 · Cited by 14

Novel mRNA therapy restores GALT protein and enzyme activity in a zebrafish model of classic galactosemia

Delnoy, B., Haskovic, M., Vanoevelen, J. M., Steinbusch, L. K. M., Vos, E. N., Knoops, K., Zimmermann, L. J. I., Noga, M. J., Lefeber, D. J., Martini, P. G. V., et al. (2022) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.12512 · Cited by 14

Disease pharmacokinetic–pharmacodynamic modelling in acute intermittent porphyria to support the development of mRNA ‐based therapies

Parra-Guillen, Z. P., Fontanellas, A., Jiang, L., Jericó, D., Martini, P. G. V., Vera‐Yunca, D., Hard, M., Guey, L. T., & Trocóniz, I. F. (2020) · British Journal of Pharmacology · doi:10.1111/bph.15040 · Cited by 14

Exploring current and emerging therapies for porphyrias

Jericó, D., Córdoba, K. M., Urigo, F., de Salamanca, R. E., Anderson, K. E., Deybach, J., Avila, M. A., & Fontanellas, A. (2024) · Liver International · doi:10.1111/liv.15979 · Cited by 12

Acute porphyrias: a German monocentric study of the biochemical, molecular genetic, and clinical data of 62 families

Bronisch, O., Stauch, T., Haverkamp, T., Beykirch, M. K., & Petrides, P. E. (2019) · Annals of Hematology · doi:10.1007/s00277-019-03831-7 · Cited by 13

Givosiran, a novel treatment for acute hepatic porphyrias

Thapar, M., Rudnick, S., & Bonkovsky, H. L. (2020) · Expert Review of Precision Medicine and Drug Development · doi:10.1080/23808993.2021.1838275 · Cited by 13

Messenger RNA rescues medium-chain acyl-CoA dehydrogenase deficiency in fibroblasts from patients and a murine model

Zhao, X., Mohsen, A.-W. A., Mihalik, S. J., Solo, K. J., Basu, S., Aliu, E., Shi, H., Kochersberger, C., Karunanidhi, A., Land, C. V., et al. (2023) · Human Molecular Genetics · doi:10.1093/hmg/ddad076 · Cited by 12

A De Novo Strategy To Improve Pharmacokinetics of Proteins from mRNA Therapeutics via Zwitterionic Polypeptide Fusion

Luozhong, S., Li, R., Tian, Z., Cao, Z., Bhashyam, D., Zhang, P., McIlhenny, K., Fang, L., McMullen, P., & Jiang, S. (2024) · Journal of the American Chemical Society · doi:10.1021/jacs.4c07903 · Cited by 12

From chemistry to genomics: A concise history of the porphyrias

Badminton, M. N., Anderson, K. E., Deybach, J., Harper, P., Sandberg, S., & Elder, G. H. (2024) · Liver International · doi:10.1111/liv.15960 · Cited by 11

Technological breakthroughs and advancements in the application of mRNA vaccines: a comprehensive exploration and future prospects

Wei, Z., Zhang, S., Wang, X., Xue, Y., Dang, S., & Zhai, J. (2025) · Frontiers in Immunology · doi:10.3389/fimmu.2025.1524317 · Cited by 16

Therapy Follows Diagnosis: Old and New Approaches for the Treatment of Acute Porphyrias, What We Know and What We Should Know

Petrides, P. E. (2022) · Diagnostics · doi:10.3390/diagnostics12071618 · Cited by 12

Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders

Ghasemzad, M., Hashemi, M., Miri-Lavasani, Z., Hossein‐Khannazer, N., Bakhshandeh, H., Gramignoli, R., Alikhani, H. K., Najimi, M., Nikeghbalian, S., & Vosough, M. (2022) · Bioengineering · doi:10.3390/bioengineering9080392 · Cited by 11

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

Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates

Córdoba, K. M., Jericó, D., Jiang, L., Collantes, M., Alegre, M., García-Ruiz, L., Manzanilla, O., Sampedro, A., Herranz, J. M., Insausti, I., et al. (2024) · Gut · doi:10.1136/gutjnl-2024-332619 · Cited by 10

Tuning the corona-core ratio of polyplex micelles for selective oligonucleotide delivery to hepatocytes or hepatic immune cells

Foo, W., Cseresnyés, Z., Rössel, C., Teng, Y., Ramoji, A., Chi, M., Hauswald, W., Huschke, S., Hoeppener, S., Popp, J., et al. (2023) · Biomaterials · doi:10.1016/j.biomaterials.2023.122016 · Cited by 12

mRNA therapeutics: Molecular mechanisms, delivery platforms, regulatory frameworks, and equitable global access

Ikrar, T., Muchsin, W., & Sophian, A. (2026) · Journal of Controlled Release · doi:10.1016/j.jconrel.2026.115019 · Cited by 10

Treatment of Experimental Autoimmune Encephalomyelitis by Sustained Delivery of Low-Dose IFN-α

Vásquez, M., Consuegra‐Fernández, M., Aranda, F., Jimenez, A., Tenesaca, S., Fernández-Sendin, M., Gomar, C., Ardaiz, N., Trani, C. A. D., Casares, N., et al. (2019) · The Journal of Immunology · doi:10.4049/jimmunol.1801462 · Cited by 9

Therapeutic nucleic acids in regenerative medicine and tissue repair

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Delivering the Message: Translating mRNA Therapy for Liver Inherited Metabolic Diseases

Gurung, S., Perocheau, D. P. B., Ghosh, R., Hart, S. L., & Baruteau, J. (2025) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.70078 · Cited by 10

Understanding Hepatic Porphyrias: Symptoms, Treatments, and Unmet Needs

Balogun, O., & Nejak‐Bowen, K. N. (2024) · Seminars in Liver Disease · doi:10.1055/s-0044-1787076 · Cited by 9

Multiple roles of haem in cystathionine β-synthase activity: implications for hemin and other therapies of acute hepatic porphyria

Badawy, A. A. ‐. (2021) · Bioscience Reports · doi:10.1042/bsr20210935 · Cited by 9

The liver as a cytokine factory working on mRNA blueprints for cancer immunotherapy

Berraondo, P., Gomis, G., & Melero, I. (2024) · Cancer Cell · doi:10.1016/j.ccell.2024.02.015 · Cited by 6

Systemic Administered mRNA as Therapy for Metabolic Diseases

Puy, H., Deybach, J., & Gouya, L. (2018) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2018.11.003 · Cited by 6

Brain ventricular enlargement in human and murine acute intermittent porphyria

Jericó, D., Luís, E. O., Cussó, L., Fernández‐Seara, M. A., Morales, X., Córdoba, K. M., Benito, M., Sampedro, A., Larriva, M., Ramírez, M. J., et al. (2020) · Human Molecular Genetics · doi:10.1093/hmg/ddaa204 · Cited by 6

bioPROTACs establish RAS as a degradable target and provide novel RAS biology insightsPreprint

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Generation of a mouse model of Primary Hyperoxaluria Type 1 via CRISPR/Cas9 mediated gene editing

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Navigating molecular therapies: The emerging role of mRNA in treating inherited metabolic diseases

Castro-Alpízar, J., Lenderink, M. J., Nieuwenhuis, E. E. S., Kooijmans, S. A. A., & Fuchs, S. A. (2025) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2025.109698 · Cited by 4

Amnio acid substitution at position 298 of human glucose-6 phosphatase-α significantly impacts its stability in mammalian cells

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RNA ‐Based Therapies for Inherited Metabolic Disorders

Reddy, S. V., Gurung, S., Ghosh, R., Mills, P. B., Baruteau, J., & Zhou, H. (2026) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.70150 · Cited by 5

Mechanistic modelling of enzyme‐restoration effects of new recombinant liver‐targeted proteins in acute intermittent porphyria

Vera‐Yunca, D., Córdoba, K. M., Parra-Guillen, Z. P., Jericó, D., Fontanellas, A., & Trocóniz, I. F. (2022) · British Journal of Pharmacology · doi:10.1111/bph.15821 · Cited by 2

When rare meets common: Treatable genetic diseases are enriched in the general psychiatric population

Sriretnakumar, V., Harripaul, R., Kennedy, J. L., & So, J. (2024) · American Journal of Medical Genetics Part A · doi:10.1002/ajmg.a.63609 · Cited by 4

New Organic Bismuth Hydrangea Materials for Electrochemical Immunoassays Targeting Carcinoembryonic Antigen

Cao, P., Tian, X., Sun, D., Wang, Z., Ding, M., Li, Y., Yang, X., Ouyang, R., & Miao, Y. (2020) · International Journal of Electrochemical Science · doi:10.20964/2020.10.06 · Cited by 3

A comprehensive analysis of the use of nucleoside analogues in RNA therapeutics

Kukushkina-Zvezdova, A. A., Zherebtsova, A. A., Telegina, D. V., Skvortsov, I. A., Ivanov, R. A., & Reshetnikov, V. V. (2026) · Journal of Translational Medicine · doi:10.1186/s12967-026-08083-4 · Cited by 3

Therapeutic approach to acute crises of hepatic porphyrias

Montes, M. G., Mataix, R. P., & Morillo, J. S. G. (2024) · Revista Clínica Española (English Edition) · doi:10.1016/j.rceng.2024.09.004 · Cited by 2

Computational disease model of phenobarbital-induced acute attacks in an acute intermittent porphyria mouse model

Vera‐Yunca, D., Serrano-Mendioroz, I., Sampedro, A., Jericó, D., Trocóniz, I. F., Fontanellas, A., & Parra-Guillen, Z. P. (2018) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2018.12.009 · Cited by 2

When awareness makes the difference: diagnosing and treating the acute hepatic porphyrias

Ventura, P. (2020) · Internal and Emergency Medicine · doi:10.1007/s11739-020-02421-0 · Cited by 2

Transamniotic Fetal Delivery of Human Alpha‐1 Antitrypsin mRNA in a Healthy Rodent Model

Naus, A. E., Moskowitzova, K., Lin, S. B., Dang, T. T., Zurakowski, D., & Fauza, D. O. (2025) · The FASEB Journal · doi:10.1096/fj.202500540 · Cited by 1

Synthesis of Precisely Modified Ribonucleic Acid to Reveal the Site-Specific Effect of Modification on Message Ribonucleic Acid Translation

Pan, Y., Zhu, C., Zhuang, Y., Li, X., Xu, R. D., Jiang, Y., Dong, Y., He, C., Liu, D., & Zhang, X. (2025) · Journal of the American Chemical Society · doi:10.1021/jacs.5c13616 · Cited by 1

Engineering lipid nanoparticle surface hydrophobicity to modulate nano-bio interface and enable tissue-selective mRNA delivery

Xu, Z., Jolly, K. J., Sankara, C. S., Mi, K., Canchola, A., Feng, H., Gillen, O. P., Heiden, K. D., Basso, K. B. G., Chou, W., et al. (2026) · Biomaterials · doi:10.1016/j.biomaterials.2026.124223 · Cited by 3

mRNA Lipid Nanoparticles for Cell Engineering in Vivo and in Vitro: Current Applications and Future Directions

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

Systemically delivered mRNA-LNPs transfect primary and secondary liver tumors

Leighton, L. J., Gee, Y. J., Madugalle, S. U., Victorova, M., Carrodus, N. L., Bridle, K. R., Howell, S. A., Liang, X., Miller, G. C., McMillan, C. L. D., et al. (2026) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2026.102989 · Cited by 3

Causal effect of porphyria biomarkers on alcohol-related hepatocellular carcinoma through Mendelian Randomization

Yang, X., Wang, S., Sun, C., & Xia, Y. (2024) · PLoS ONE · doi:10.1371/journal.pone.0299536 · Cited by 1

Abordaje terapéutico de las crisis agudas de porfirias hepáticas

Montes, M. G., Mataix, R. P., & Morillo, J. S. G. (2024) · Revista Clínica Española · doi:10.1016/j.rce.2024.07.004 · Cited by 1

Gene Therapy for Inherited Metabolic Diseases

Longo, N., & Lai, K. (2022) · Springer eBooks · doi:10.1007/978-3-030-94510-7_8 · Cited by 1

ARN mensajero (mRNA), una molécula con potencial aplicación terapéutica y preventiva

Rosas, G., Bobes, R. J., Cervantes‐Torres, J. B., Sciutto, E., & Fragoso, G. (2022) · Inventio, la génesis de la cultura universitaria en Morelos · doi:10.30973/inventio/2022.18.44/8

Efficient delivery of mRNA-LNPs in primary and secondary liver cancerPreprint

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Acute Intermittent Porphyria: Novel Etiologic and Pathogenic Therapies Based on RNA Transfer to the Liver

Prìeto, J., & González‐Aseguinolaza, G. (2019) · Hepatology · doi:10.1002/hep.30678

Criminalidade económica: entre o crime de colarinho branco e o crime de colarinho azul, existirá o crime de colarinho cinzento?

Ferreira, J. M. D. S. C. (2013) · Portuguese National Funding Agency for Science, Research and Technology (RCAAP Project by FCT)

Synthetic mRNA Gene Therapies and Hepatotropic Non-viral Vectors for the Treatment of Chronic HBV Infections

Kairuz, D., Singh, P., Smith, T. S., Arbuthnot, P. B., Ely, A., & Bloom, K. M. (2022) · RNA technologies · doi:10.1007/978-3-031-08415-7_8

Exogenous RNAs as Potential Pharmacological Agents

Okovitiy, S. V., & Шустов, Е. Б. (2022) · Journal Biomed · doi:10.33647/2074-5982-18-3-118-121

The Orphan Liver Disease

Khan, R. S., & Newsome, P. N. (2022) · In clinical practice · doi:10.1007/978-3-031-10012-3_14

Papel de SLU7 en la diferenciación hepática y en la protección del hígado frente al daño: regulación de HNF4A

Gárate‐Rascón, M. (2022) · Deposito Adademico Digital Universidad De Navarra (University of Navarra) · doi:10.15581/10171/65009

Análise do efeito do ácido 5-aminolevulínico sobre o perfil transcricional de HepG2

Menezes, P. R. (2020) · doi:10.11606/d.87.2020.tde-21092023-121318

De la investigación básica a la práctica clínica: ARN de interferencia

Barreda‐Sánchez, M., & Guillén‐Navarro, E. (2023) · Medicina Clínica · doi:10.1016/j.medcli.2023.05.030

Fisiopatología de los ataques en las porfirias hepáticas agudas

D’Avola, D. (2023) · Medicina Clínica · doi:10.1016/j.medcli.2023.05.029

Tailoring the bioactivities of IL-12 for cancer immunotherapy with mRNA-based technology: from immunocytokines to armored T cell transfer

Di-Trani, C. A. (. A. (2023) · Deposito Adademico Digital Universidad De Navarra (University of Navarra) · doi:10.15581/10171/67876

Research Progress on mRNA Based Protein Replacement Therapy for Inborn Errors of Metabolism

李, 京. (2025) · Advances in Clinical Medicine · doi:10.12677/acm.2025.154899

Development Of mRNA Vaccines And Therapeutics Beyond Covid-19

Yadav, A., Dwivedi, A., & Darwhekar, G. N. (2025) · European journal of parenteral & pharmaceutical sciences · doi:10.37521/ejpps30211

Animal Models of Porphyria with Hepatic Involvement

Balogun, O., & Nejak‐Bowen, K. N. (2025) · Seminars in Liver Disease · doi:10.1055/a-2677-6806

mRNA-based therapies: current progress and future prospects for treating kidney disease

Song, Y., & Su, H. (2025) · Clinical and Experimental Nephrology · doi:10.1007/s10157-025-02785-7

Future of Regenerative Hepatology

Martínez-García, J., Blanes-Rodríguez, Á., Barbancho, S. M., Viartola, M. S., & Baptista, P. M. (2026) · Springer eBooks · doi:10.1007/978-3-032-10997-2_23

Preclinical development and evaluation of Uricase mRNA-loaded lipid nanoparticles for the treatment of hyperuricemia

Zhang, J., Wen, J., Li, T., Xu, J., Ma, J., Hong, M. J., & Li, W. (2026) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2026.108029

Co-Designing mRNA Cargo and Lipid Nanoparticles across Sequence Function, Intracellular Trafficking, Biodistribution, and Manufacturabil

Ali, H., Farooq, M., Shah, U., Khan, B., & Malik, S. (2026) · Pharmacophore · doi:10.51847/4ylxcn8kfc

A biomimetic delivery strategy for achieving dual-specific lipid nanoparticle targeting

Duan, Y., Zhang, F., Guerrero, E. D., Sung, Y., Tian, Z., Wang, X., Basak, P., Chatterjee, S., Farbiak, L., & Siegwart, D. J. (2026) · Cell Biomaterials · doi:10.1016/j.celbio.2026.100554

Advances and Challenges in Nanodelivery Systems for RNA Vaccines

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