Protocol for Efficient Generation and Characterization of Adeno-Associated Viral Vectors

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Gao, G.-P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., & Wilson, J. M. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182412299 · Cited by 1,520 · see citing works

Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

Xiao, X., Li, J., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.3.2224-2232.1998 · Cited by 1,381 · see citing works

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

Zolotukhin, S., Byrne, B. J., Mason, E. G., Zolotukhin, I., Potter, M., Chesnut, K. A., Summerford, C., Samulski, R. J., & Muzyczka, N. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300938 · Cited by 1,317 · see citing works

Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors

Grimm, D., Kern, A., Rittner, K., & Kleinschmidt, J. A. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.18-2745 · Cited by 700 · see citing works

The family Parvoviridae

Cotmore, S. F., Agbandje‐McKenna, M., Chiorini, J. A., Mukha, D. V., Pintel, D. J., Qiu, J., Söderlund‐Venermo, M., Tattersall, P. J., Tijssen, P., Gatherer, D., et al. (2013) · Archives of Virology · doi:10.1007/s00705-013-1914-1 · Cited by 693 · see citing works

Adeno-associated virus vectors can be efficiently produced without helper virus

Matsushita, T., Elliger, S., Elliger, C., Podsakoff, G., Villarreal, L., Kurtzman, G., Iwaki, Y., & Colosi, P. (1998) · Gene Therapy · doi:10.1038/sj.gt.3300680 · Cited by 507 · see citing works

Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.

Hermonat, P. L., & Muzyczka, N. (1984) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.81.20.6466 · Cited by 480 · see citing works

Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo

Wang, Z., Ma, H.-I., Li, J., Sun, L., Zhang, J., & Xiao, X. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302133 · Cited by 413 · see citing works

Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6

Grimm, D., Kay, M. A., & Kleinschmidt, J. A. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00095-9 · Cited by 344 · see citing works

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α ₁ -Antitrypsin: Interim Results

Flotte, T. R., Trapnell, B. C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A. T., Sandhaus, R. A., McElvaney, N. G., et al. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.053 · Cited by 335 · see citing works

Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector

Grieger, J. C., Soltys, S. M., & Samulski, R. J. (2015) · Molecular Therapy · doi:10.1038/mt.2015.187 · Cited by 326 · see citing works

Transfection of mammalian cells using linear polyethylenimine is a simple and effective means of producing recombinant adeno-associated virus vectors

Reed, S. E., Staley, E. M., Mayginnes, J. P., Pintel, D. J., & Tullis, G. E. (2006) · Journal of Virological Methods · doi:10.1016/j.jviromet.2006.07.024 · Cited by 251 · see citing works

Endotoxin Limits in Formulations for Preclinical Research

Malyala, P., & Singh, M. (2007) · Journal of Pharmaceutical Sciences · doi:10.1002/jps.21152 · Cited by 206 · see citing works

A Human Parvovirus, Adeno-Associated Virus, as a Eucaryotic Vector: Transient Expression and Encapsidation of the Procaryotic Gene for Chloramphenicol Acetyltransferase

Tratschin, J. D., West, M. H., Sandbank, T., & Carter, B. J. (1984) · Molecular and Cellular Biology · doi:10.1128/mcb.4.10.2072 · Cited by 202 · see citing works

The Assembly-Activating Protein Promotes Capsid Assembly of Different Adeno-Associated Virus Serotypes

Sonntag, F., Köther, K., Schmidt, K., Weghofer, M., Raupp, C., Nieto, K., Kuck, A., Gerlach, B., Böttcher, B., Müller, O. J., et al. (2011) · Journal of Virology · doi:10.1128/jvi.05359-11 · Cited by 184 · see citing works

Purification of Recombinant Adeno-Associated Virus by Iodixanol Gradient Ultracentrifugation Allows Rapid and Reproducible Preparation of Vector Stocks for Gene Transfer in the Nervous System

Hermens, W. T. J. M. C., ter Brake, O., Dijkhuizen, P. A., Sonnemans, M. A. F., Grimm, D., Kleinschmidt, J. A., & Verhaagen, J. (1999) · Human Gene Therapy · doi:10.1089/10430349950017563 · Cited by 180 · see citing works

Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models

Rapti, K., Louis-Jeune, V., Kohlbrenner, E., Ishikawa, K., Ladage, D., Zolotukhin, S., Hajjar, R. J., & Weber, T. (2012) · Molecular Therapy · doi:10.1038/mt.2011.177 · Cited by 170 · see citing works

Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors

Müller, O. J., Leuchs, B., Pleger, S. T., Grimm, D., Franz, W. M., Katus, H. A., & Kleinschmidt, J. A. (2006) · Cardiovascular Research · doi:10.1016/j.cardiores.2005.12.017 · Cited by 156 · see citing works

High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap

Conway, J. E., Rhys, C. A., Zolotukhin, I., Zolotukhin, S., Muzyczka, N., Hayward, G. S., & Byrne, B. J. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300937 · Cited by 154 · see citing works

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material

Lock, M., McGorray, S., Auricchio, A., Ayuso, E., Beecham, E. J., Blouin-Tavel, V., Bosch, F., Bose, M., Byrne, B. J., Caton, T., et al. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.223 · Cited by 131 · see citing works

Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

Gaudet, D., de Wal, J., Tremblay, K., Déry, S., van Deventer, S. J. H., Freidig, A. P., Brisson, D., & Méthot, J. (2010) · Atherosclerosis Supplements · doi:10.1016/j.atherosclerosissup.2010.03.004 · Cited by 122 · see citing works

Adeno-Associated Virus Type 2 Protein Interactions: Formation of Pre-Encapsidation Complexes

Dubielzig, R., King, J., Weger, S., Kern, A., & Kleinschmidt, J. A. (1999) · Journal of Virology · doi:10.1128/jvi.73.11.8989-8998.1999 · Cited by 105 · see citing works

Rescue and replication of adeno-associated virus type 2 as well as vector DNA sequences from recombinant plasmids containing deletions in the viral inverted terminal repeats: Selective encapsidation of viral genomes in progeny virions

Wang, X. S., Ponnazhagan, S., & Srivastava, A. (1996) · Journal of Virology · doi:10.1128/jvi.70.3.1668-1677.1996 · Cited by 104 · see citing works

Scalable Recombinant Adeno-Associated Virus Production Using Recombinant Herpes Simplex Virus Type 1 Coinfection of Suspension-Adapted Mammalian Cells

Thomas, D. L., Wang, L., Niamke, J., Liu, J., Kang, W., Scotti, M. M., Ye, G., Veres, G., & Knop, D. R. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.004 · Cited by 97 · see citing works

An efficient rHSV-based complementation system for the production of multiple rAAV vector serotypes

Kang, W., Wang, L., Harrell, H., Liu, J., Thomas, D., Mayfield, T. L., Scotti, M. M., Ye, G. J., Veres, G., & Knop, D. R. (2008) · Gene Therapy · doi:10.1038/gt.2008.158 · Cited by 56 · see citing works

Economized large‐scale production of high yield of rAAV for gene therapy applications exploiting baculovirus expression system

Negrete, A., Yang, L. C., Mendez, A. F., Levy, J. R., & Kotin, R. M. (2007) · The Journal of Gene Medicine · doi:10.1002/jgm.1092 · Cited by 54 · see citing works

Process optimization of large-scale production of recombinant adeno-associated vectors using dielectric spectroscopy

Negrete, A., Esteban, G., & Kotin, R. M. (2007) · Applied Microbiology and Biotechnology · doi:10.1007/s00253-007-1030-9 · Cited by 48 · see citing works

Production of recombinant adeno-associated vectors using two bioreactor configurations at different scales

Negrete, A., & Kotin, R. M. (2007) · Journal of Virological Methods · doi:10.1016/j.jviromet.2007.05.020 · Cited by 44 · see citing works

Transfection-free and scalable recombinant AAV vector production using HSV/AAV hybrids

Booth, M. J., Mistry, A., Li, X., Thrasher, A. J., & Coffin, R. S. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302226 · Cited by 43 · see citing works

35. High Titer Recombinant Adeno-Associated Virus Production Using Replication Deficient Herpes Simplex Viruses Type I

(2003) · Molecular Therapy · doi:10.1016/s1525-0016(16)40477-6 · Cited by 5 · see citing works

Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies

Gimpel, A. L., Katsikis, G., Sha, S., Maloney, A. J., Hong, M. S., Nguyen, T. N. T., Wolfrum, J., Springs, S. L., Sinskey, A. J., Manalis, S. R., et al. (2021) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2021.02.010 · Cited by 185 · see citing works

High-throughput mapping of a whole rhesus monkey brain at micrometer resolution

Xu, F., Shen, Y. X., Ding, L., Yang, C., Tan, H., Wang, H., Zhu, Q., Xu, R., Wu, F., Xiao, Y., et al. (2021) · Nature Biotechnology · doi:10.1038/s41587-021-00986-5 · Cited by 137 · see citing works

Restriction of essential amino acids dictates the systemic metabolic response to dietary protein dilution

Yap, Y. W., Rusu, P. M., Chan, A. Y., Fam, B. C., Jungmann, A., Solon‐Biet, S. M., Barlow, C. K., Creek, D. J., Huang, C., Schittenhelm, R. B., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-16568-z · Cited by 118 · see citing works

Liver alanine catabolism promotes skeletal muscle atrophy and hyperglycaemia in type 2 diabetes

Okun, J. G., Rusu, P. M., Chan, A. Y., Wu, Y., Yap, Y. W., Sharkie, T., Schumacher, J., Schmidt, K. V., Roberts‐Thomson, K. M., Russell, R., et al. (2021) · Nature Metabolism · doi:10.1038/s42255-021-00369-9 · Cited by 117 · see citing works

Secretome Analysis of Cardiomyocytes Identifies PCSK6 (Proprotein Convertase Subtilisin/Kexin Type 6) as a Novel Player in Cardiac Remodeling After Myocardial Infarction

Kuhn, T. C., Knobel, J., Burkert-Rettenmaier, S., Li, X., Meyer, I. S., Jungmann, A., Sicklinger, F., Backs, J., Lasitschka, F., Müller, O. J., et al. (2020) · Circulation · doi:10.1161/circulationaha.119.044914 · Cited by 84 · see citing works

Sources of off-target expression from recombinase-dependent AAV vectors and mitigation with cross-over insensitive ATG-out vectors

Fischer, K. B., Collins, H. K., & Callaway, E. M. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1915974116 · Cited by 69 · see citing works

The lipid-droplet-associated protein ABHD5 protects the heart through proteolysis of HDAC4

Jebessa, Z. H., Shanmukha, K. D., Dewenter, M., Lehmann, L., Xu, C., Schreiter, F. C., Siede, D., Gong, X.-M., Worst, B. C., Federico, G., et al. (2019) · Nature Metabolism · doi:10.1038/s42255-019-0138-4 · Cited by 63 · see citing works

A human cell atlas of the pressure-induced hypertrophic heart

Nicin, L., Schroeter, S. M., Glaser, S.-F., Bruening, R. S., Pham, M. D., Hille, S. S., Yekelchyk, M., Kattih, B., Abplanalp, W. T., Tombor, L. S., et al. (2022) · Nature Cardiovascular Research · doi:10.1038/s44161-022-00019-7 · Cited by 59 · see citing works

AKAP18δ Anchors and Regulates CaMKII Activity at Phospholamban-SERCA2 and RYR

Carlson, C. R., Aronsen, J. M., Bergan-Dahl, A., Moutty, M. C., Lunde, M., Lunde, P., Jarstadmarken, H. O., Wanichawan, P., Pereira, L., Kolstad, T. R., et al. (2021) · Circulation Research · doi:10.1161/circresaha.120.317976 · Cited by 54 · see citing works

Reduction of A-to-I RNA editing in the failing human heart regulates formation of circular RNAs

Kokot, K. E., Kneuer, J. M., John, D., Rebs, S., Möbius-Winkler, M. N., Erbe, S., Müller, M., Andritschke, M., Gaul, S., Sheikh, B. N., et al. (2022) · Basic Research in Cardiology · doi:10.1007/s00395-022-00940-9 · Cited by 49 · see citing works

Preclinical evidence for the therapeutic value of TBX5 normalization in arrhythmia control

Rathjens, F. S., Blenkle, A., Iyer, L. M., Renger, A., Syeda, F., Noack, C., Jungmann, A., Dewenter, M., Toischer, K., El‐Armouche, A., et al. (2020) · Cardiovascular Research · doi:10.1093/cvr/cvaa239 · Cited by 48 · see citing works

Genetic Insights into Primary Restrictive Cardiomyopathy

Brodehl, A., & Gerull, B. (2022) · Journal of Clinical Medicine · doi:10.3390/jcm11082094 · Cited by 48 · see citing works

Nephron segment-specific gene expression using AAV vectors

Asico, L. D., Cuevas, S., Ma, X., José, P. A., Armando, I., & Konkalmatt, P. R. (2018) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2018.01.169 · Cited by 44 · see citing works

Multiplexed chemogenetics in astrocytes and motoneurons restore blood–spinal cord barrier in ALS

Alami, N. O., Tang, L., Wiesner, D., Commisso, B., Bayer, D., Weishaupt, J. H., Dupuis, L., Wong, P. C., Baumann, B., Wirth, T., et al. (2020) · Life Science Alliance · doi:10.26508/lsa.201900571 · Cited by 39 · see citing works

Single-Use Capture Purification of Adeno-Associated Viral Gene Transfer Vectors by Membrane-Based Steric Exclusion Chromatography

Marichal‐Gallardo, P., Börner, K., Pieler, M., Sonntag-Buck, V., Obr, M., Bejarano, D. A., Wolff, M. W., Kräusslich, H., Reichl, U., & Grimm, D. (2021) · Human Gene Therapy · doi:10.1089/hum.2019.284 · Cited by 38 · see citing works

Stage-dependent remodeling of projections to motor cortex in ALS mouse model revealed by a new variant retrograde-AAV9

Commisso, B., Ding, L., Varadi, K., Gorges, M., Bayer, D., Boeckers, T. M., Ludolph, A. C., Kassubek, J., Müller, O. J., & Roselli, F. (2018) · eLife · doi:10.7554/elife.36892 · Cited by 32 · see citing works

Alginate hydrogel polymers enable efficient delivery of a vascular-targeted AAV vector into aortic tissue

Remes, A. K., Basha, D. I., Puehler, T., Borowski, C., Hille, S. S., Kummer, L., Wagner, A. H., Hecker, M., Soethoff, J. P., Lutter, G., et al. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.02.017 · Cited by 33 · see citing works

The E3 ubiquitin ligase HectD3 attenuates cardiac hypertrophy and inflammation in mice

Rangrez, A. Y., Borlepawar, A., Schmiedel, N., Deshpande, A., Remes, A. K., Kumari, M., Bernt, A., Christen, L., Helbig, A. O., Jungmann, A., et al. (2020) · Communications Biology · doi:10.1038/s42003-020-01289-2 · Cited by 32 · see citing works

Disturbed flow–induced Gs-mediated signaling protects against endothelial inflammation and atherosclerosis

Nakayama, A., Albarrán-Juárez, J., Liang, G., Roquid, K. A., Iring, A., Tonack, S., Chen, M., Müller, O. J., Weinstein, L. S., & Offermanns, S. (2020) · JCI Insight · doi:10.1172/jci.insight.140485 · Cited by 29 · see citing works

XBP1s-EDEM2 Prevents the Onset and Development of HFpEF by Ameliorating Cardiac Lipotoxicity

Fonseka, O., Raja, R., Ross, C., Gare, S. R., Zhang, J., Hille, S. S., King, K., Ruiz‐Velasco, A., Kaur, N., Chen, X., et al. (2025) · Circulation · doi:10.1161/circulationaha.124.072194 · Cited by 28 · see citing works

Deletion of NoxO1 limits atherosclerosis development in female mice

Buchmann, G., Schürmann, C., Warwick, T., Schulz, M. H., Spaeth, M., Müller, O. J., Schröder, K., Jo, H., Weißmann, N., & Brandes, R. P. (2020) · Redox Biology · doi:10.1016/j.redox.2020.101713 · Cited by 26 · see citing works

Accurate Quantification of AAV Vector Genomes by Quantitative PCR

de la Cámara, C. M.-F., McClements, M. E., & MacLaren, R. E. (2021) · Genes · doi:10.3390/genes12040601 · Cited by 25 · see citing works

Cardiac Fibroblast Growth Factor 23 Excess Does Not Induce Left Ventricular Hypertrophy in Healthy Mice

Leifheit‐Nestler, M., Wagner, M., Richter, B., Piepert, C., Eitner, F., Böckmann, I., Vogt, I., Grund, A., Hille, S. S., Foinquinos, A., et al. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.745892 · Cited by 24 · see citing works

AAV-mediated cardiac gene transfer of wild-type desmin in mouse models for recessive desminopathies

Ruppert, T., Heckmann, M. B., Rapti, K., Schultheis, D., Jungmann, A., Katus, H. A., Winter, L., Frey, N., Clemen, C. S., Schröder, R., et al. (2020) · Gene Therapy · doi:10.1038/s41434-020-0147-7 · Cited by 16 · see citing works

The activity of glyoxylase 1 is regulated by glucose-responsive phosphorylation on Tyr136

Cortizo, F. G., Pfaff, D. H., Wirth, A., Schlotterer, A., Medert, R., Morgenstern, J., Weber, T., Hammes, H., Fleming, T., Nawroth, P. P., et al. (2021) · Molecular Metabolism · doi:10.1016/j.molmet.2021.101406 · Cited by 14 · see citing works

CITED4 gene therapy protects against maladaptive cardiac remodeling after ischemia/reperfusion injury in mice

Lerchenmüller, C., Hastings, M. H., Rabolli, C. P., Betge, F., Roshan, M., Liu, L. X., Liu, X., Hess, C., Roh, J. D., Platt, C., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.07.018 · Cited by 14 · see citing works

Cardiac tumour necrosis factor receptor-associated factor 7 mediates the ubiquitination of apoptosis signal-regulating kinase 1 and aggravates cardiac hypertrophy

Che, Y., Liu, Y., Wang, Z., Feng, Y.-Z., Xia, H., Yuan, Y., Zhou, H., Qiu, H.-L., Hu, M., Wang, S., et al. (2024) · Cardiovascular Research · doi:10.1093/cvr/cvae217 · Cited by 14 · see citing works

Various effects of AAV9-mediated βARKct gene therapy on the heart in dystrophin-deficient (mdx) mice and δ-sarcoglycan-deficient (Sgcd-/-) mice

Bauer, R., Enns, H., Jungmann, A., Leuchs, B., Volz, C., Schinkel, S., Koch, W. J., Raake, P. W. J., Most, P., Katus, H. A., et al. (2018) · Neuromuscular Disorders · doi:10.1016/j.nmd.2018.12.006 · Cited by 14 · see citing works

Nuclear eNOS Interacts With and S -Nitrosates ADAR1 to Modulate Type I Interferon Signaling and Endothelial Function

Zhou, X., Kuenne, C. T., Günther, S., Wittig, I., Güven, B., Boutguetait, D., Lagos, F. D., Sachs, N., Mäegdefessel, L., Müller, O. J., et al. (2025) · Circulation · doi:10.1161/circulationaha.125.074889 · Cited by 13 · see citing works

LncRNA MDRL Mitigates Atherosclerosis through miR-361/SQSTM1/NLRP3 Signaling

You, L., Zheng, Y., Yang, J., Hou, Q., Wang, L., Zhang, Y., Zhao, C., & Xie, R. (2022) · Mediators of Inflammation · doi:10.1155/2022/5463505 · Cited by 13 · see citing works

Novel Nongenetic Murine Model of Hyperglycemia and Hyperlipidemia-Associated Aggravated Atherosclerosis

Gaul, S., Shahzad, K., Medert, R., Gadi, I., Maeder, C., Schumacher, D., Wirth, A., Ambreen, S., Fatima, S., Boeckel, J., et al. (2022) · Frontiers in Cardiovascular Medicine · doi:10.3389/fcvm.2022.813215 · Cited by 12 · see citing works

Analytical ultracentrifugation sedimentation velocity for the characterization of recombinant adeno-associated virus vectors sub-populations

Saleün, S., Mas, C., Roy, A. L., Penaud‐Budloo, M., Adjali, O., Blouin, V., & Ebel, C. (2023) · European Biophysics Journal · doi:10.1007/s00249-023-01650-3 · Cited by 12 · see citing works

AFM-based nanoindentation indicates an impaired cortical stiffness in the AAV-PCSK9DY atherosclerosis mouse model

Achner, L., Klersy, T., Fels, B., Reinberger, T., Schmidt, C. X., Groß, N., Hille, S. S., Müller, O. J., Aherrahrou, Z., Kusche‐Vihrog, K., et al. (2022) · Pflügers Archiv - European Journal of Physiology · doi:10.1007/s00424-022-02710-x · Cited by 13 · see citing works

In Vivo Delivery of Cassettes Encoding Anti-HBV Primary MicroRNAs Using an Ancestral Adeno-Associated Viral Vector

Mnyandu, N., Arbuthnot, P. B., & Maepa, M. B. (2020) · Methods in molecular biology · doi:10.1007/978-1-0716-0290-4_10 · Cited by 10 · see citing works

Ligand-activated RXFP1 gene therapy ameliorates pressure overload-induced cardiac dysfunction

Sasipong, N., Schlegel, P., Wingert, J., Lederer, C., Meinhardt, E., Ziefer, A., Schmidt, C., Rapti, K., Thoeni, C. E., Frey, N., et al. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.04.010 · Cited by 10 · see citing works

Development of an AAV9-RNAi-mediated silencing strategy to abrogate TRPM4 expression in the adult heart

Medert, R., Jungmann, A., Hildebrand, S., Busch, M., Grimm, D., Flockerzi, V., Müller, O. J., Most, P., Schumacher, D., & Freichel, M. (2021) · Pflügers Archiv - European Journal of Physiology · doi:10.1007/s00424-021-02521-6 · Cited by 9 · see citing works

Restored autophagy is protective against PAK3-induced cardiac dysfunction

Ruiz‐Velasco, A., Raja, R., Chen, X., Ganenthiran, H., Kaur, N., Alatawi, N. H. O., Miller, J. M., Abouleisa, R., Ou, Q., Zhao, X., et al. (2023) · iScience · doi:10.1016/j.isci.2023.106970 · Cited by 9 · see citing works

Regnase-1 overexpression as a therapeutic approach of Marfan syndrome

Noormalal, M., Schmiedel, N., Bozoglu, T. M., Matzen, A., Hille, S. S., Basha, D. I., Shetty, P. M. V., Wolf, A. A., Zaradzki, M., Arif, R., et al. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101163 · Cited by 8 · see citing works

Prolylcarboxypeptidase Alleviates Hypertensive Cardiac Remodeling by Regulating Myocardial Tissue Angiotensin II

Nguyen, B. Y., Zhou, F., Binder, P., Liu, W., Hille, S. S., Luo, X., Zi, M., Zhang, H., Adamson, A. D., Ahmed, F. Z., et al. (2023) · Journal of the American Heart Association · doi:10.1161/jaha.122.028298 · Cited by 8 · see citing works

Incidence of microvascular dysfunction is increased in hyperlipidemic mice, reducing cerebral blood flow and impairing remote memory

Hernandez Torres, L. D., Rezende, F., Peschke, E., Will, O., Hövener, J.-B., Spiecker, F., Özorhan, Ü., Lampe, J., Stölting, I., Aherrahrou, Z., et al. (2024) · Frontiers in Endocrinology · doi:10.3389/fendo.2024.1338458 · Cited by 8 · see citing works

Accurate and Rapid Sequence Analysis of Adeno-Associated Virus Plasmids by Illumina Next-Generation Sequencing

Saveliev, A., Liu, J., Li, M., Hirata, L., Latshaw, C., Zhang, J., & Wilson, J. M. (2018) · Human Gene Therapy Methods · doi:10.1089/hgtb.2018.037 · Cited by 8 · see citing works

Dietary Essential Amino Acid Restriction Promotes Hyperdipsia via Hepatic FGF21

Rusu, P. M., Chan, A. Y., Heikenwälder, M. F., Müller, O. J., & Rose, A. J. (2021) · Nutrients · doi:10.3390/nu13051469 · Cited by 8 · see citing works

Cerebellar pathology contributes to neurodevelopmental deficits in spinal muscular atrophy

Gerstner, F., Wittig, S., Menedo, C., Ruwald, S., Carlini, M. J., Vankova, A., Sowoidnich, L., Martín-López, G., Dreilich, V., Alonso-Collado, A., et al. (2025) · Brain · doi:10.1093/brain/awaf336 · Cited by 7 · see citing works

Cryptotanshinone Attenuated Pathological Cardiac Remodeling In Vivo and In Vitro Experiments

Li, W.-J., Yan, H., Zhou, Z.-Y., Zhang, N., Ding, W., Liao, H.-H., & Tang, Q.-Z. (2023) · Oxidative Medicine and Cellular Longevity · doi:10.1155/2023/4015199 · Cited by 8 · see citing works

AAV library screening identifies novel vector for efficient transduction of human aorta

Schröder, L. C., Hüttermann, L., Remes, A. K., Voran, J. C., Hille, S. S., Sommer, W., Lutter, G., Warnecke, G., Frank, D., Schade, D., et al. (2024) · Gene Therapy · doi:10.1038/s41434-024-00511-8 · Cited by 6 · see citing works

Progress and application of cell membrane engineering in living cell therapy

Qiu, X., Sun, Y., Gao, J., & Jiang, X. (2025) · Biomaterials Science · doi:10.1039/d5bm00120j · Cited by 6 · see citing works

Endothelial ZBTB16: a molecular shield against cardiac aging

Stilz, K. A., Leonard, V. E., Morales, D. R., Glaser, S.-F., Larcher, V., Jurado, M. R., Malacarne, P. F., Manickam, N., Tombor, L. S., Abplanalp, W. T., et al. (2025) · European Heart Journal · doi:10.1093/eurheartj/ehaf1063 · Cited by 5 · see citing works

Phosphoproteomics-directed manipulation reveals SEC22B as a hepatocellular signaling node governing metabolic actions of glucagon

Wu, Y., Foollee, A., Chan, A. Y., Hille, S. S., Hauke, J., Challis, M. P., Johnson, J. L., Yaron, T. M., Mynard, V., Aung, O. H., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-52703-w · Cited by 5 · see citing works

The anti-atherosclerotic effect of chronic AT1 receptor blocker treatment also depends on the ACE2/Ang(1−7)/Mas axis

Klersy, T., Achner, L., Fels, B., Rezende, F., Lopez, M., Alenina, N. V., Spiecker, F., Stölting, I., Häuser, W., Reinberger, T., et al. (2025) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2025.117990 · Cited by 4 · see citing works

RGS3L allows for an M2 muscarinic receptor-mediated RhoA-dependent inotropy in cardiomyocytes

Levay, M. K., Krobert, K. A., Vogt, A., Ahmad, A. N., Jungmann, A., Neuber, C., Pasch, S., Hansen, A., Müller, O. J., Lutz, S., et al. (2022) · Basic Research in Cardiology · doi:10.1007/s00395-022-00915-w · Cited by 4 · see citing works

Amlodipine improves aortic dysfunction in atherosclerotic mice

Rasch, S. M., Fels, B., Schmalohr, A., Torres, L. D. H., Peschke, E., Will, O., Hövener, J., Achner, L., Klersy, T., Spiecker, F., et al. (2026) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2026.119073 · Cited by 3 · see citing works

High-throughput proteo-genomic identification of functional endothelial cell microproteins encoded by non-canonical small open reading framesPreprint

Siragusa, M., Graumann, J., Kuenne, C. T., Günther, S., Jeratsch, S., Güven, B., Raheja, M., Süsser, S., Talyan, S., Bezuneh, B., et al. (2024) · Research Square · doi:10.21203/rs.3.rs-4194943/v1 · Cited by 3 · see citing works

Directed Evolution of AAV9 for Efficient Gene Expression in Cardiomyocytes In Vitro and In Vivo

Hüttermann, L., Schröder, L. C., Shetty, P. M. V., Jonker, T., Hille, S. S., Remes, A. K., Matzen, A., Boender, A., Grimm, D., Frank, D., et al. (2025) · Human Gene Therapy · doi:10.1089/hum.2024.126 · Cited by 2 · see citing works

Real-time genome imaging of host interactions in adeno-associated virus genome release

Bustamante-Jaramillo, L. F., Yue, L., Fingal, J., Rydell, G. E., Johansson, M., Edreira, T., Müller, O. J., Hille, S. S., Müller, M., Gallardo, F., et al. (2025) · iScience · doi:10.1016/j.isci.2025.112624 · Cited by 2 · see citing works

Selective Enrichment of Full Capsids of Adeno-Associated Virus

Bal, V., Wolfrum, J. M., Barone, P. W., Springs, S. L., Sinskey, A. J., Kotin, R. M., & Braatz, R. D. (2025) · ACS Nano · doi:10.1021/acsnano.5c00566 · Cited by 2 · see citing works

Large-scale mapping of the MCH network in ALS mice reveals the vulnerability of dopaminergic and GABAergic neurons in zona incerta

Scekic‐Zahirovic, J., Antonucci, S., Wiesner, D., Ebner, C., Hajj, H. E., Aousji, O., Halablab, K., Fan, Y. B., Zelaya, A., Yartas, G., et al. (2026) · Acta Neuropathologica Communications · doi:10.1186/s40478-026-02231-z · Cited by 1 · see citing works

FGFR1/KLB-MFG-E8 Maintains Microvascular Integrity and Adaptive Cardiac Remodeling

Gare, S. R., Alatawi, N. H. O., Chen, X., Zhang, J., Ross, C. A., Fonseka, O., Han, Y., Kaur, N., Ruiz‐Velasco, A., Zhao, X., et al. (2026) · Circulation Research · doi:10.1161/circresaha.125.327950 · Cited by 1 · see citing works

Titin cleavage impairs cardiac mechanical connectivity to drive diastolic failure and fibrosisPreprint

Freundt, J. K., Hartmann, P., Loescher, C. M., Unger, A., Koser, F., Klotz, A. J., Wachsmuth, L., Hille, S. S., Door, M. M., Helfen, A., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.04.21.649485 · Cited by 1 · see citing works

CAMK2D causes heart failure in RBM20 cardiomyopathyPreprint

van den Hoogenhof, M. M. G., Durán, J., Britto‐Borges, T., Kemmling, E., Konrad, L., Lennermann, D. C., Hartmann, J., Schraft, L., Kornienko, J., Bock, T., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.05.23.655557 · Cited by 1 · see citing works

Immunomodulatory endothelial cells contribute to T cell recruitment and activation through antigen presentation on MHC class II

Cartura, M., Aliraj, B., Szymanski, W. G., Ferrario, G., Rezende, F., Güven, B., Bleckwehl, T., Dimmeler, S., Müller, O. J., Jo, H., et al. (2026) · doi:10.21248/gups.99203 · Cited by 1 · see citing works

Genomic G quadruplexes regulate mRNA splicingPreprint

Mauz, V., Britto‐Borges, T., Merkel, T. S., Gjerga, E., Hartmann, J., Snaebjornsson, M. T., Schreiter, F. C., Oeing, C. U., Sticht, C., Sossalla, S. T., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2025.12.15.693900 · Cited by 1 · see citing works

Restriction of essential amino acids dictates the systemic response to dietary protein dilutionPreprint

Yap, Y. W., Rusu, P. M., Chan, A. Y., Fam, B. C., Jungmann, A., Solon‐Biet, S. M., Barlow, C. K., Creek, D. J., Huang, C., Schittenhelm, R. B., et al. (2019) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/848143

An endocrine-hepato-muscular metabolic cycle links skeletal muscle atrophy and hyperglycemia in type 2 diabetesPreprint

Okun, J. G., Rusu, P. M., Chan, A. Y., Yap, Y. W., Sharkie, T., Schumacher, J., Schmidt, K. V., Zota, A., Hille, S. S., Jungmann, A., et al. (2020) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2020.05.25.115709

A novel non-genetic murine model of hyperglycemia and hyperlipidemia-associated aggravated atherosclerosisPreprint

Gaul, S., Shahzad, K., Medert, R., Gadi, I., Maeder, C., Schumacher, D., Wirth, A., Ambreen, S., Fatima, S., Boeckel, J., et al. (2021) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2021.11.11.468191

A Coherent Receiver for QPSK and SQPSK Signals

Alem, W., & Weber, C. (1978) · UA Campus Repository (The University of Arizona)

Molecular mechanisms of TBX5-related conduction disorders

Rathjens, F. S. (2021) · doi:10.53846/goediss-8451

Cardiac TNF Receptor-Associated Factor 7 Mediates the Ubiquitination of Apoptosis Signal-Regulating Kinase 1 and Aggravates Cardiac hypertrophyPreprint

Che, Y., Xia, H., Wang, Z., Feng, Y., Liu, Y., Yuan, Y., Zhou, H., Wang, S., & Tang, Q. (2022) · Research Square · doi:10.21203/rs.3.rs-1952573/v1

LncRNA Bigheart trans-activates gene expression in a feed forward mechanism that facilitates calcineurin-NFAT signaling in myocardial hypertrophyPreprint

Mangraviti, N., Rühle, F., Kinet, V., Hegenbarth, J.-C., Dirkx, E., Spanò, G., Majo, F. D., Peppas, P., Martens, L., Gandhi, S., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.12.11.571094

PAK3 Exacerbates Cardiac Lipotoxicity via SREBP1c in Obesity CardiomyopathyShort Title: PAK3 provokes cardiac lipotoxicity

Chen, X., Ruiz‐Velasco, A., Zou, Z., Hille, S. S., Ross, C., Fonseka, O., Gare, S. R., Alatawi, N. H. O., Raja, R., Zhang, J., et al. (2024) · doi:10.2337/figshare.26510980.v1

PAK3 Exacerbates Cardiac Lipotoxicity via SREBP1c in Obesity CardiomyopathyShort Title: PAK3 provokes cardiac lipotoxicity

Chen, X., Ruiz‐Velasco, A., Zou, Z., Hille, S. S., Ross, C., Fonseka, O., Gare, S. R., Alatawi, N. H. O., Raja, R., Zhang, J., et al. (2024) · doi:10.2337/figshare.26510980

Cerebellar pathology contributes to neurodevelopmental deficits in spinal muscular atrophyPreprint

Simon, C. M., Gerstner, F., Wittig, S., Menedo, C., Ruwald, S., Carlini, M. J., Vankova, A., Sowoidnich, L., Martín-López, G., Dreilich, V., et al. (2025) · Research Square · doi:10.21203/rs.3.rs-6819992/v1

Endothelial expression of ZBTB16 protects against cardiac agingPreprint

Stilz, K. A., Leonard, V. E., Morales, D. R., Glaser, S.-F., Larcher, V., Jurado, M. R., Malacarne, P. F., Manickam, N., Tombor, L. S., Abplanalp, W. T., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.10.08.681100

CAMK2-NR4A1 signaling initiates metabolic substrate switching to induce heart failure with reduced ejection fractionPreprint

Saadatmand, A., Pepin, M. E., Chen, Z., Hartmann, J., Sun, Q., Dewenter, M., Nazir, S., Paluvai, H., Hagenmüller, M., Haberkorn, U., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.10.16.682731

High Phosphate Load Induces De Novo Formation of Tertiary Lymphoid Structures in the Kidney

Weingärtner, N., Richter, B., Walles, F., Kapanadze, T. G., Schmitz, J., Bräsen, J. H., Limbourg, F. P., Haffner, D., & Leifheit‐Nestler, M. (2025) · The FASEB Journal · doi:10.1096/fj.202500968r

A nutrient-hormone axis pivots an energy trade-off between resting thermogenesis and movement expenditure

Rusu, P. M., Aung, O. H., Chan, A. Y., Wu, Y., Cleofe, M. A. S., Trewin, A. J., Giri, S., Foollee, A., flores, S., Lemus, M. B., et al. (2026) · Journal of sport and health science/Journal of Sport and Health Science · doi:10.1016/j.jshs.2026.101132

CAMK2D causes heart failure in mice with RBM20 cardiomyopathy

van den Hoogenhof, M. M. G., Durán, J., Britto‐Borges, T., Sequeira, V., Kemmling, E., Konrad, L., Schreiter, F. C., Lennermann, D. C., Hartmann, J., Schraft, L., et al. (2026) · Nature Cardiovascular Research · doi:10.1038/s44161-026-00818-2

Selective titin cleavage disrupts cardiac mechanical homeostasis to drive heart failure and fibrosis

Freundt, J. K., Hartmann, P., Loescher, C. M., Unger, A., Koser, F., Klotz, A. J., Wildschütz, L., Wachsmuth, L., Hille, S. S., Door, M. M., et al. (2026) · Nature Cardiovascular Research · doi:10.1038/s44161-026-00829-z · Cited by 1 · see citing works

Aortic asprosin overexpression does not ameliorate disease pathophysiology in a murine model of Marfan syndrome

Shetty, P. M. V., Matzen, A., Hille, S. S., Michalewski, S., Witthaus, H., Noormalal, M., Marcous, F., Morcos, Y. A. T., Bozoglu, T. M., Sommer, W., et al. (2026) · Scientific Reports · doi:10.1038/s41598-026-59187-2

Efficient and High‐Resolution Separation of Partial, Empty, and Full AAV Capsids by Optimized Iodixanol Density Gradient Ultracentrifugation

Fang, S., Wu, J., Shen, Y., Qi, D., Ni, Z., & FengHua, W. A. N. G. (2026) · Biotechnology Journal · doi:10.1002/biot.70319

Protocol for efficient generation and characterization of adeno-associated viral (AAV) vectors

Jungmann, A., Leuchs, B., Katus, H. A., Rommelaere, J., & Müller, O. J. (2017) · Human Gene Therapy Methods · doi:10.1089/hum.2017.192 · Cited by 6 · see citing works

Production of recombinant adeno-associated viral vectors for in vitro and in vivo use

Choi, V. W., Asokan, A., Haberman, R. A., & Samulski, R. J. (2007) · Current Protocols in Molecular Biology · doi:10.1002/0471142727.mb1625s78 · Cited by 52 · see citing works

Production of Recombinant Adeno‐Associated Viral Vectors

Choi, V. W., Asokan, A., Haberman, R. A., & Samulski, R. J. (2007) · Current Protocols in Human Genetics · doi:10.1002/0471142905.hg1209s53 · Cited by 171 · see citing works

Production and characterization of adeno-associated viral vectors

Grieger, J. C., Choi, V. W., & Samulski, R. J. (2006) · Nature Protocols · doi:10.1038/nprot.2006.207 · Cited by 567 · see citing works

Production of first generation adenoviral vectors for preclinical protocols: Amplification, purification and functional titration

Armendáriz‐Borunda, J., Bastidas‐Ramírez, B. E., Sandoval-Rodriguez, A. S., González-Cuevas, J., Gómez‐Meda, B. C., & Garcia-Bañuelos, J. J. (2011) · Journal of Bioscience and Bioengineering · doi:10.1016/j.jbiosc.2011.07.018 · Cited by 23 · see citing works