Manufacturing and characterizing AAV-based vectors for use in clinical studies

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163

Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions

Summerford, C., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.2.1438-1445.1998 · Cited by 1,409

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

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 1,321

Use of Adeno-Associated Virus as a General Transduction Vector for Mammalian Cells

Muzyczka, N. (1992) · Current topics in microbiology and immunology · doi:10.1007/978-3-642-75608-5_5 · Cited by 756

Cd8(+) T-Cell Responses to Adeno-Associated Virus Capsid in Humans

Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E. J., Ragni, M. V., Manno, C. S., Sommer, J., Jiang, H., et al. (2007) · Nature Medicine · doi:10.1038/nm1549 · Cited by 748

Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression

Samulski, R. J., Chang, L., & Shenk, T. E. (1989) · Journal of Virology · doi:10.1128/jvi.63.9.3822-3828.1989 · Cited by 742

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 701

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

Insect Cells as a Factory to Produce Adeno-Associated Virus Type 2 Vectors

Urabe, M., Ding, C., & Kotin, R. M. (2002) · Human Gene Therapy · doi:10.1089/10430340260355347 · Cited by 457

The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

Kyostio-Moore, S., Berthelette, P., Piraino, S., Sookdeo, C., Nambiar, B., Jackson, R., Burnham, B. L., O’Riordan, C. R., Cheng, S. H., & Armentano, D. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.6 · Cited by 18

Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection

Nambiar, B., Sookdeo, C., Berthelette, P., Jackson, R., Piraino, S., Burnham, B. L., Nass, S., Souza, D. W., O’Riordan, C. R., Vincent, K. A., et al. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.124 · Cited by 17

Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors

Whiteley, Z., Massaro, G., Gkogkos, G., Gavriilidis, A., Waddington, S. N., Rahim, A. A., & Craig, D. Q. M. (2023) · Nanoscale · doi:10.1039/d2nr06401d · Cited by 16

Multi‐omics kinetic analysis of recombinant adeno‐associated virus production by plasmid transfection of HEK293 cells

Lu, M., Lee, Z., & Hu, W. (2024) · Biotechnology Progress · doi:10.1002/btpr.3428 · Cited by 17

Macrocyclic polyamine [12]aneN 3 modified triphenylamine-pyrazine derivatives as efficient non-viral gene vectors with AIE and two-photon imaging properties

Ma, L., Liu, M., Liu, X., Sun, W. F., Lu, Z., Gao, Y., & He, L. (2020) · Journal of Materials Chemistry B · doi:10.1039/d0tb00321b · Cited by 16

A Novel and Simplified Anion Exchange Flow‐Through Polishing Approach for the Separation of Full From Empty Adeno‐Associated Virus Capsids

Meierrieks, F., Weltken, A., Pflanz, K. B., Pickl, A., Graf, B., & Wolff, M. W. (2024) · Biotechnology Journal · doi:10.1002/biot.202400430 · Cited by 16

Developments in the treatment of hemophilia B: focus on emerging gene therapy

Gray, J. T., Cancio, M., Reiss, U. M., Nathwani, A. C., & Davidoff, A. M. (2013) · The Application of Clinical Genetics · doi:10.2147/tacg.s31928 · Cited by 17

Surface Modified Nano-Electrospray Needles Improve Sensitivity for Native Mass Spectrometry

Kostelic, M. M., Hsieh, C., Sanders, H. M., Zak, C. K., Ryan, J. P., Baker, E. S., Aspinwall, C. A., & Marty, M. T. (2022) · Journal of the American Society for Mass Spectrometry · doi:10.1021/jasms.2c00087 · Cited by 15

Drug product Formulation and Fill/Finish Manufacturing Process Considerations for AAV-Based Genomic Medicines

Som, M., Gikanga, B., Kanapuram, V., & Yadav, S. (2024) · Journal of Pharmaceutical Sciences · doi:10.1016/j.xphs.2024.03.024 · Cited by 13

In vitro cell subtype-specific transduction of adeno-associated virus in mouse and marmoset retinal explant culture

Baba, Y., Satoh, S., Otsu, M., Sasaki, E., Okada, T., & Watanabe, S. (2012) · Biochimie · doi:10.1016/j.biochi.2012.08.010 · Cited by 13

Nonimmunogenetic Viral Capsid Carrier with Cancer Targeting Activity

Lee, B., Jo, E., Yoon, H. Y., Yoon, C. J., Lee, H., Kwon, K. C., Kim, T. W., & Lee, J. (2018) · Advanced Science · doi:10.1002/advs.201800494 · Cited by 13

Enhancement of rAAV titers via inhibition of the interferon signaling cascade in transfected HEK293 suspension cultures

Kahlig, C., Moser, S., Mičutková, L., Grillari, J., Kraus, B., & Bort, J. A. H. (2024) · Biotechnology Journal · doi:10.1002/biot.202300672 · Cited by 12

Size-exclusion chromatography as a multi-attribute method for process and product characterization of adeno-associated virus

Mulagapati, S. H. R., Parupudi, A., Witkos, T. M., Bond, N., Chen, X., Linke, T. A., Xi, G., Schmelzer, A. E., & Wei, X. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101382 · Cited by 12

Large-Scale Clinical Manufacturing of AAV Vectors for Systemic Muscle Gene Therapy

Clément, N. (2019) · Springer eBooks · doi:10.1007/978-3-030-03095-7_15 · Cited by 12

Viral vector-mediated gene therapy for opioid use disorders

Wang, T., Zhu, X., Yi, H., Gu, J., Liu, S., Izenwasser, S., Lemmon, V., Roy, S., & Hao, S. (2021) · Experimental Neurology · doi:10.1016/j.expneurol.2021.113710 · Cited by 12

Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine Zippers

Thadani, N. N., Yang, J., Moyo, B., Lee, C. M., Chen, M. Y., Bao, G., & Suh, J. (2020) · ACS Synthetic Biology · doi:10.1021/acssynbio.9b00341 · Cited by 12

DNA Released by Adeno-Associated Virus Strongly Alters Capsid Aggregation Kinetics in a Physiological Solution

Jarand, C. W., Baker, K., Petroff, M., Jin, M., & Reed, W. F. (2024) · Biomacromolecules · doi:10.1021/acs.biomac.4c00027 · Cited by 12

CRISPR/Cas9 Gene Editing: A Novel Approach Towards Alzheimer's Disease Treatment

Tripathi, S., Sharma, Y., Rane, R. A., & Kumar, D. (2024) · CNS & Neurological Disorders - Drug Targets · doi:10.2174/0118715273283786240408034408 · Cited by 11

A Changing World in Gene Therapy Research: Exciting Opportunities for Medical Advancement and Biosafety Challenges

Eisenman, D., Debold, S., & Riddle, J. (2021) · Applied Biosafety · doi:10.1089/apb.2021.0020 · Cited by 11

Large-Scale Transient Transfection of Suspension Mammalian Cells for VLP Production

Cervera, L., & Kamen, A. (2017) · Methods in molecular biology · doi:10.1007/978-1-4939-7312-5_10 · Cited by 11

Gene Therapy

Thorne, B., Takeya, R. K., Vitelli, F., & Swanson, X. (2017) · Advances in biochemical engineering, biotechnology · doi:10.1007/10_2016_53 · Cited by 11

Preferential exclusion chromatography as a capture step for extracellular AAV harvest from adherent and suspension productions

Leskovec, M., Raspor, A., Fujs, V., Mihevc, A., & Štrancar, A. (2023) · Electrophoresis · doi:10.1002/elps.202300038 · Cited by 11

A simple and sensitive differential digestion method to analyze adeno-associated virus residual host cell proteins by LC-MS

Hu, Y., Hu, M., Ye, X., Wu, Z., Kang, J., Wong, C., Palackal, N. T., Qiu, H., & Li, N. (2024) · Journal of Pharmaceutical and Biomedical Analysis · doi:10.1016/j.jpba.2024.116009 · Cited by 10

Immunogenicity risk assessment of empty capsids present in adeno-associated viral vectors using predictive innate immune responses

Jarvi, N. L., Hofman, K., Venkatesh, A., Gorecki, E., & Balu‐Iyer, S. V. (2024) · Journal of Pharmaceutical Sciences · doi:10.1016/j.xphs.2024.09.006 · Cited by 10

AAV Vectors for Efficient Gene Delivery to Rodent Hearts

López-Gordo, E., Kohlbrenner, E., Katz, M. G., & Weber, T. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9139-6_19 · Cited by 10

Analysis of Host Cell Proteins in AAV Products with ProteoMiner Protein Enrichment Technology

Zhang, S., Xiao, H., & Li, N. (2024) · Analytical Chemistry · doi:10.1021/acs.analchem.3c03884 · Cited by 9

MicroRNA‐based recombinant AAV vector assembly improves efficiency of suicide gene transfer in a murine model of lymphoma

Khan, N., Cheemadan, S., Saxena, H., Bammidi, S., & Jayandharan, G. R. (2020) · Cancer Medicine · doi:10.1002/cam4.2935 · Cited by 8

C3 Transferase Gene Therapy for Continuous RhoA Inhibition

Santiago-Lopez, A. J., Gutekunst, C., & Gross, R. E. (2018) · Methods in molecular biology · doi:10.1007/978-1-4939-8612-5_19 · Cited by 9

Single pass tangential flow filtration (SPTFF) for concentration and purification of AAV clarified cell lysate

Chaubal, A. S., Yehl, C. J., Afzal, M. A., Wang, L., & Zydney, A. L. (2025) · Journal of Membrane Science · doi:10.1016/j.memsci.2025.124362 · Cited by 8

Characterization of Recombinant Chimpanzee Adenovirus C68 Low and High-Density Particles: Impact on Determination of Viral Particle Titer

Mullins, E. K., Powers, T. W., Zobel, J., Clawson, K. M., Barnes, L. F., Draper, B. E., Zou, Q., Binder, J. J., Dai, S., Zhang, K., et al. (2021) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2021.753480 · Cited by 8

Insights into Adeno-Associated Virus Capsid Charge Heterogeneity

Wu, J., Draper, B. E., Jarrold, M. F., & Heger, C. D. (2025) · Analytical Chemistry · doi:10.1021/acs.analchem.5c03104 · Cited by 7

Adeno-associated virus analysis by size exclusion chromatography within 3 minutes using short bio-inert columns made with 3 µm particles operated at high flowrates

Song, J., & Farkas, T. (2024) · Journal of Chromatography A · doi:10.1016/j.chroma.2024.464684 · Cited by 7

Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization

Liu, X., Jean-Gilles, R., Baginski, J., Cai, C., Yan, R., Zhang, L., Lance, K., van der Loo, J. C. M., & Davidson, B. L. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101298 · Cited by 7

Utilizing adeno‐associated virus as a vector in treating genetic disorders or human cancers

Shih, F., Chang, H.-H., & Wang, Y. (2024) · IUBMB Life · doi:10.1002/iub.2896 · Cited by 7

Clinical Design and Analysis Strategies for the Development of Gene Therapies: Considerations for Quantitative Drug Development in the Age of Genetic Medicine

McIntosh, A., Sverdlov, O., Yu, L., & Kaufmann, P. (2021) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.2224 · Cited by 7

Integrating the affinity and anion-exchange purification of AAV8: how the selective enrichment of full capsids at the affinity capture step enhances the downstream processing of gene therapies

Shastry, S., Minzoni, A., Hurst, M. R., Collazo, C., Barbieri, E., Chu, W., Taris, J. E., Gilbert, P., Major, C., Daniele, M. A.-A., et al. (2025) · Journal of Chromatography A · doi:10.1016/j.chroma.2025.466557 · Cited by 6

Corneal Neovascularization: Pathogenesis, Current Insights and Future Strategies

Muller, E., Feinberg, L. J., Woronkowicz, M., & Roberts, H. W. (2026) · Biology · doi:10.3390/biology15020136 · Cited by 8

Adjuvant effect of B domain of staphyloccocal protein A displayed on the surface of hepatitis B virus capsid

Kim, H. J., Ahn, K.-Y., Bae, K., Lee, J., Sim, S. J., & Lee, J. (2015) · Biotechnology and Bioengineering · doi:10.1002/bit.25716 · Cited by 6

Advanced Therapies: Clinical, Non-clinical and Quality Considerations

Hoogendoorn, K. H. (2019) · Springer eBooks · doi:10.1007/978-3-030-00710-2_17 · Cited by 6

Enrichment of Full AAV2 Using Multicolumn Countercurrent Solvent Gradient Purification (MCSGP)

Müller, J. M., Müllerová, T., Tobler, D., Hauri, D., Plieninger, R., Higuchi, Y., Takahashi, R., Vogg, S., Müller‐Späth, T., & Villiger, T. K. (2025) · Biotechnology and Bioengineering · doi:10.1002/bit.29036 · Cited by 7

Stem cell and gene therapies for leukodystrophies

Lin, W., Zhang, M., Zheng, S., & Lian, Q. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101527 · Cited by 7

Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF

Thomas, S. P., Spinelli, M. M., Rghei, A. D., Lopes, J. A., Zielinska, N., Mcleod, B. M., Pei, Y.-L., Zhang, W., Thébaud, B., Karimi, K., et al. (2024) · BMC Biotechnology · doi:10.1186/s12896-024-00853-6 · Cited by 7

Native Digestion and Shotgun Proteomics for Host Cell Protein Profiling of Adeno-Associated Viruses.

Lodge, J., Huang, L., Lian, Z., Qian, J., & Tian, Y. (2024) · Analytical Chemistry · doi:10.1021/acs.analchem.4c00893 · Cited by 6

The manufacture of AAV for gene therapy applications using a closed, semi-automated hollow-fiber bioreactor

Soula, A., Leseigneur, F., Anwar, A., Ozdoganoglu, B., Gurung, J., Bhatti, H. S., Guenat, J., Bazot, Q., Sayed, M. E. E., Ricardo, C. P., et al. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101496 · Cited by 5

Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy

Cagin, U. (2021) · Advances in experimental medicine and biology · doi:10.1007/978-3-030-55035-6_15 · Cited by 5

AAV Vector-Based Gene Therapy, Progress and Current Challenges

Kuranda, K., & Mingozzi, F. (2017) · Springer eBooks · doi:10.1007/978-3-319-53457-2_5 · Cited by 5

Challenges in development and authorisation of gene therapy products

Алексеевич, С. А., Авдеева, Ж. И., Витальевич, Г. Д., Khantimirova, L. M., Guseva, S. G., & Меркулов, В. А. (2022) · Biological Products Prevention Diagnosis Treatment · doi:10.30895/2221-996x-2022-22-1-6-22 · Cited by 5

Recent Advances in Canine Medicine

Fonseca‐Alves, C. E. (2022) · Veterinary medicine and science · doi:10.5772/intechopen.100825 · Cited by 6

Molecular mechanisms in podocytopathies: finding suitable targets for a new era of glomerular gene therapy

Tavakolidakhrabadi, N., Ding, W. Y., Welsh, G. I., & Saleem, M. A. (2025) · Clinical Kidney Journal · doi:10.1093/ckj/sfaf332 · Cited by 4

Process Development for the Production and Purification of Adeno-Associated Virus (AAV)2 Vector using Baculovirus-Insect Cell Culture System

Nasimuzzaman, M., Villaveces, S., van der Loo, J. C. M., & Alla, S. (2022) · Journal of Visualized Experiments · doi:10.3791/62829 · Cited by 4

Clinical practice guidelines for gene therapy to treat hereditary hearing loss

Qi, J., Tan, F., Zhang, L., Lü, L., Wang, H., Li, W., Liu, W., Fu, X., He, Z., Ding, X., et al. (2024) · Interdisciplinary medicine · doi:10.1002/inmd.20240008 · Cited by 4

The role of pharmaceutical formulation development in clinical trials of new medicinal products

Owczarek, B., & Markowicz-Piasecka, M. (2026) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2026.115101 · Cited by 3

Codon-Optimized and de novo–Synthesized E-Selectin/AAV2 Dose–Response Study for Vascular Regeneration Gene Therapy

Voza, F. A., Byrne, B. J., Ortiz, Y. Y., Li, Y., Le, N., Osafo, L., Ribieras, A. J., Shao, H., Huerta, C. T., Wei, Y., et al. (2024) · Annals of Surgery · doi:10.1097/sla.0000000000006436 · Cited by 3

A Hitchhiker’s Guide to the Selection of Viral Vectors for Optogenetic Studies

Thompson, K. R., & Towne, C. L. (2017) · Neuromethods · doi:10.1007/978-1-4939-7417-7_1 · Cited by 4

Present Situation of Viral Vector Manufacturing and Ways to Overcome Potential Barriers in View of the Routine Large Scale Production and Use of Viral Vectors

Merten, O.-W. (2017) · Current Trends in Biomedical Engineering & Biosciences · doi:10.19080/ctbeb.2017.07.555704 · Cited by 3

Stability of Recombinant Mosaic Adeno-Associated Virus Vector rAAV/DJ/CAG at Different Temperature Conditions

Bieńkowska, A., & Małecki, M. (2021) · Journal of Biomedical Nanotechnology · doi:10.1166/jbn.2021.3183 · Cited by 3

CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector production

O'Driscoll, E., Arora, S., Lang, J. F., Davidson, B. L., & Shalem, O. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101408 · Cited by 5

Stability and structural evolution of double-stranded DNA molecules under high pressures: A molecular dynamics study

Herrera-Velarde, S., Villanueva-Valencia, J. R., Mendoza-Espinosa, P., & Castañeda-Priego, R. (2023) · Frontiers in Physics · doi:10.3389/fphy.2023.1076787 · Cited by 2

Manufacturing of Viral Gene Therapies

Ramirez, G. A., & Gasmi, M. (2021) · International Ophthalmology Clinics · doi:10.1097/iio.0000000000000362 · Cited by 2

Optimization of Internally Deleted Dystrophin Constructs

Reza, M., Laval, S. H., Roos, A., Carr, S., & Lochmüller, H. (2016) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.026 · Cited by 2

Manufacturing and Control of Viral‐Vector Vaccines

Chavda, V. P., Vaghela, D. A., Barman, D., Newar, A., & Ahmed, N. (2025) · doi:10.1002/9781394271566.ch8 · Cited by 2

Degradation Study of Different Brands of Rosuvastatin Calcium Tablet in Bangladesh Using UV Spectrophotometer

Nahar, J., Al-Amin, M., & Sharna, S. M. (2020) · International Journal of Innovative Science and Research Technology (IJISRT) · doi:10.38124/ijisrt20jul097 · Cited by 1

Bio-Production of Adeno-Associated Virus for Gene Therapy

Donohue, N., Keogh, N., Boi, S., & Barron, N. (2021) · Cell engineering · doi:10.1007/978-3-030-79871-0_11 · Cited by 1

Chromatographic Purification of Viral Vectors for Gene Therapy Applications

Kearney, A. (2023) · Methods in molecular biology · doi:10.1007/978-1-0716-3362-5_4 · Cited by 1

Implementing a robust platform analytical procedure for measuring adeno-associated virus vector genome titer

Powers, T. W., Sloan, C. D. K., Stano, D., Evans, B. P., Liu, K., Mariani, S., Campbell, J. A., Lerch, T. F., & Mo, J. J. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101381 · Cited by 1

Chronologically distributed transfection improves AAV2 and AAV2/8 capsid filling and reveals assembly schedule divergence

Chen, Q., Lee, C. H., Whitfield, R., & Nesbeth, D. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101610 · Cited by 1

Significant Enhancement of Adeno‐Associated Virus Vector Yield for Gene Therapy Using Multidimensional Optimization Strategies

Shen, W., Chen, S., Li, Z., & Yu, W. (2025) · The FASEB Journal · doi:10.1096/fj.202502611rr · Cited by 1

Postępy w leczeniu chorych na hemofilie

Windyga, J. (2015) · Hematologia · doi:10.5603/hem.2015.0021 · Cited by 1

Stem Cell Based Exosomes: Are They Effective in Disease or Health?

Kucukguven, M. B., & Çelebi‐Saltik, B. (2021) · Advances in experimental medicine and biology · doi:10.1007/5584_2021_630 · Cited by 1

Reversed Phase-Liquid Chromatography for Recombinant AAV Genome Integrity Assessment

Gstöttner, C., Hutanu, A., Boon, S., Raducanu, A., Richter, K., Haindl, M., Ruppert, R., & Domínguez‐Vega, E. (2023) · Analytical Chemistry · doi:10.1021/acs.analchem.3c00222 · Cited by 1

Gene Therapy Challenges in Arthritis

Denys, A., Thiolat, A., Boissier, M., & Natach, B. (2011) · InTech eBooks · doi:10.5772/19300

Gene Therapy Trials in Hemophilia A and B

High, K. A. (2014) · Wiley eBooks · doi:10.1002/9781118398258.ch41

Expanding AAV cargo capacity for gene therapy of Stargardt disease

Trapani, I. (2014)

AAV2 vermittelte Transduktion von porcinem Myokard im Transplantationsmodell

Schulz, A. (2009) · Elektronische Hochschulschriften der LMU München (Ludwig-Maximilians-Universität München) · doi:10.5282/edoc.10823

Novel strategies to improve viral gene targeting and therapeutic liver repopulation in vivo

Paulk, N. K. (2012) · doi:10.6083/m4k935jk

Gene therapy in mouse models of spinal muscular atrophy :||using SMN gene replacement to address biological questions

Glascock, J. (2013) · MOspace Institutional Repository (University of Missouri) · doi:10.32469/10355/44658

Gene Therapy Trials in Hemophilia A and B

High, K. A., & Wright, J. F. (2010) · Wiley eBooks · doi:10.1002/9781444318555.ch36

Inner Ear Gene Therapy in Mouse Models of Genetic Hearing Loss

Choi, J. W. (2021) · Korean Journal of Otorhinolaryngology - Head and Neck Surgery · doi:10.3342/kjorl-hns.2020.00836

Gene and Cell Therapy Products

Working, P. K. (2014) · doi:10.1002/9781118873922.ch24

Parvovirus Vectors: The Future of Gene Therapy

Gupta, M. (2022) · Veterinary medicine and science · doi:10.5772/intechopen.105085

Therapy for neurodegenerative Diseases and expectations

Jiang, Y. (2023) · Highlights in Science Engineering and Technology · doi:10.54097/hset.v36i.6282

Characterization of Adeno-Associated Virus (AAV) Gene Therapy Products

Rathore, A. S., Auclair, J. R., & Kaur, A. (2023) · LCGC North America · doi:10.56530/lcgc.na.st3379l8

Adeno-Associated Virus Analysis by Size Exclusion Chromatography within 3 Minutes Using Short Bio-Inert Columns Made with 3 Μm Particles Operated at High FlowratesPreprint

Song, J., & Farkas, T. (2023) · SSRN Electronic Journal · doi:10.2139/ssrn.4658165

Advanced Therapy Medicinal Products: Clinical, Non-clinical, and Quality Considerations

Mastrobattista, E., Doevendans, E., van Til, N. P., Kemp, V., de Vrij, J., & Hoogendoorn, K. H. (2024) · Springer eBooks · doi:10.1007/978-3-031-30023-3_14

The current status and future trends in immunotoxicogenomics

Bhat, A., Shah, R., Sharma, M., Mahajan, K., & Kumar, R. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-18502-1.00013-4

A Droplet Digital PCR Assay for Quantification of Bacteriophage Viral Vector Titer and Purity

Voorhees, P. J., Ponek, R. M., Liu, J. D., & Lai, S. K. (2026) · Annals of Biomedical Engineering · doi:10.1007/s10439-026-04117-6

High-Resolution Characterization of rAAV Genomes for Vector Quality Using Long-Read SequencingPreprint

Wu, C.-J., Liu, H., Carlile, T. M., Lin, D., Zhang, B., Zhang, W., & Diallo, A. B. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.10.05.680573

Modulation of the intraocular immune environment by viral gene transfer in mouse models of uveitis

Chu, C. J. (2015) · UCL Discovery (University College London)

Additional file 1 of Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF

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