CpG-depleted adeno-associated virus vectors evade immune detection

Cited by (254)

Items 101–200 of 254

1. Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors
   Helena Costa Verdera, Carmen Unzu, Erika Valeri, Sahil Adriouch, Gloria González‐Aseguinolaza, Federico Mingozzi, Anna Kajaste‐Rudnitski · 2023 · Human Gene Therapy
2. Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns
   Glenn F. Pierce · 2020 · Haemophilia
3. AAV-Mediated Gene Delivery to the Liver: Overview of Current Technologies and Methods
   Brett Palaschak, Roland W. Herzog, David M. Markusic · 2019 · Methods in molecular biology
4. Advances in the Development and the Applications of Nonviral, Episomal Vectors for Gene Therapy
   Grace E. Mulia, Virgínia Picanço‐Castro, Eleana F. Stavrou, Aglaia Athanassiadou, Marxa Leão Figueiredo · 2021 · Human Gene Therapy
5. Delivery of genome editing tools: A promising strategy for HPV-related cervical malignancy therapy
   Shahin Aghamiri, Sam Talaei, Soheil Roshanzamiri, Farshid Zandsalimi, Elnaz Fazeli, Mansur Aliyu, Omid Kheiry Avarvand, Zahra Ebrahimi, Mahsa Keshavarz‐Fathi, Hossein Ghanbarian · 2020 · Expert Opinion on Drug Delivery
6. TLR9-Activating CpG-B ODN but Not TLR7 Agonists Triggers Antibody Formation to Factor IX in Muscle Gene Transfer
   Butterfield, John S.S., Biswas, Moanaro, Shirley, Jamie L., Kumar, Sandeep R.P., Sherman, Alexandra, Terhorst, Cox, Ling, Chen, Herzog, Roland W. · 2019 · Human Gene Therapy Methods
7. The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution
   Artyom Kachanov, Anastasiya Kostyusheva, Sergey Brezgin, Ivan Karandashov, N. I. Ponomareva, Andrey Tikhonov, Alexander N. Lukashev, Vadim S. Pokrovsky, Andrey A. Zamyatnin, Alessandro Parodi et al. · 2024 · Medicinal Research Reviews
8. Modifying immune responses to adeno-associated virus vectors by capsid engineering
   Bentler, Martin, Hardet, Romain, Ertelt, Moritz, Rudolf, Daniela, Kaniowska, Dorota, Schneider, Andreas, Vondran, Florian W.R., Schoeder, Clara T., Delphin, Marion, Lucifora, Julie et al. · 2023 · Molecular Therapy - Methods & Clinical Development
9. Establishment of two quantitative nested qPCR assays targeting the human EPO transgene
   Elmo W. I. Neuberger, Irene C. Perez, Caroline Le Guiner, Dirk Moser, Tobias Ehlert, M. Allais, Philippe Moullier, Perikles D. Simon, Richard O. Snyder · 2016 · Gene Therapy
10. Gene targeting as a therapeutic avenue in diseases mediated by the complement alternative pathway
    Anna K. Dreismann, Thomas M Hallam, Lawrence C. S. Tam, Calvin V Nguyen, Jane P. Hughes, Scott Ellis, Claire Louise Harris · 2022 · Immunological Reviews
11. Obstacles and future of gene therapy for hemophilia
    Arruda, Valder R, Samelson-Jones, Ben J · 2015 · Expert Opinion on Orphan Drugs
12. Ocular Inflammation and Treatment Emergent Adverse Events in Retinal Gene Therapy
    Mehta, Neesurg, Robbins, Deborah A., Yiu, Glenn · 2021 · International Ophthalmology Clinics
13. Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?
    Wei Zhan, Manish Muhuri, Phillip W.L. Tai, Guangping Gao · 2021 · Frontiers in Immunology
14. Synthetic Biology—Toward Therapeutic Solutions
    Viktor Haellman, Martin Fussenegger · 2015 · Journal of Molecular Biology
15. Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH
    Seemin Seher Ahmed, Hillard L. Rubin, Minglun Wang, Deiby Faulkner, Arnold Sengooba, Serena Dollive, Nancy Avila, Jeff L. Ellsworth, Diana Lamppu, Maria Lobikin et al. · 2020 · Molecular Therapy — Methods & Clinical Development
16. Promise and Progress of an HIV-1 Cure by Adeno-Associated Virus Vector Delivery of Anti-HIV-1 Biologics
    Matthew R. Gardner · 2020 · Frontiers in Cellular and Infection Microbiology
17. Overcoming Immunological Challenges Limiting Capsid-Mediated Gene Therapy With Machine Learning
    Anna Z. Wec, Kathy S. Lin, Jamie C. Kwasnieski, Sam Sinai, Jeff Gerold, Eric D. Kelsic · 2021 · Frontiers in Immunology
18. Hepatotoxicity in Adeno-Associated Viral Vector Gene Therapy
    Barath Jagadisan, Anil Kumar Dhawan · 2023 · Current Hepatology Reports
19. Developing Immunologically Inert Adeno-Associated Virus (AAV) Vectors for Gene Therapy: Possibilities and Limitations
    Ruchita Selot, Sangeetha Hareendran, Giridhara Rao Jayandharan · 2014 · Current Pharmaceutical Biotechnology
20. Gene Therapeutic Strategies for Peripheral Artery Disease and New Opportunities Provided by Adeno-Associated Virus Vectors
    Levon Michael Khachigian, Ramon Lee Varcoe, Tuisku Suoranta, Nihay Laham-Karam, Seppo Ylä‐Herttuala · 2023 · Arteriosclerosis Thrombosis and Vascular Biology
21. TLR9-independent CD8+ T cell responses in hepatic AAV gene transfer through IL-1R1-MyD88 signaling
    Kumar, Sandeep R.P., Biswas, Moanaro, Cao, Di, Arisa, Sreevani, Muñoz-Melero, Maite, Lam, Anh K., Piñeros, Annie R., Kapur, Reuben, Kaisho, Tsuneyasu, Kaufman, Randal J. et al. · 2024 · Molecular Therapy
22. AAV genome modification for efficient AAV production
    Walaa Asaad, Polina Volos, Denis O. Maksimov, Elena M. Khavina, Andrei A. Deviatkin, Olga N. Mityaeva, Pavel Volchkov · 2023 · Heliyon
23. Gene Transfer Therapy for Neurodevelopmental Disorders
    Can Özlü, Rachel M. Bailey, Sarah E. Sinnett, Kimberly Goodspeed · 2021 · Developmental Neuroscience
24. Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for Ex-Vivo Gene Editing
    Amanda M. Dudek, Matthew Hebden Porteus · 2021 · Frontiers in Immunology
25. The immune response to the fVIII gene therapy in preclinical models
    Patel, Seema R., Lundgren, Taran S., Spencer, H. Trent, Doering, Christopher B. · 2020 · Frontiers in Immunology
26. Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies
    Kumar, Sandeep R.P., Duan, Dongsheng, Herzog, Roland W. · 2023 · Human Gene Therapy
27. Strategies to improve safety profile of AAV vectors
    Tuisku Suoranta, Nihay Laham-Karam, Seppo Ylä‐Herttuala · 2022 · Frontiers in Molecular Medicine
28. Engineering challenges and translational opportunities in emerging gene delivery platforms
    Yifan Ma, Shiyan Dong, Annette Wu, Seong Dong Jeong, Andrew Stephen Lee, Wen Jiang, Betty Y.S. Kim · 2026 · Nature Biomedical Engineering
29. Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors
    Julie A. Hutt, Basel T. Assaf, Brad N. Bolon, Joy A. Cavagnaro, Elizabeth J. Galbreath, Branka Grubor, Lisa M. Kattenhorn, Annette Romeike, Laurence O. Whiteley · 2021 · Toxicologic Pathology
30. Clinical and Translational Landscape of Viral Gene Therapies
    Alexandra Yudaeva, Anastasiya Kostyusheva, Artyom Kachanov, Sergey Brezgin, Natalia Ponomareva, Alessandro Parodi, Vadim S. Pokrovsky, Alexander N. Lukashev, Vladimir Chulanov, Dmitry S. Kostyushev · 2024 · Cells
31. Neuro-Immuno-Gene- and Genome-Editing-Therapy for Alzheimer’s Disease: Are We There Yet?
    Sudhanshu P. Raikwar, Ramasamy Thangavel, Iuliia Dubova, Mohammad Ejaz Ahmed, Govindhasamy Pushpavathi Selvakumar, Duraisamy Kempuraj, Smita A. Zaheer, Shankar S. Iyer, Asgar Zaheer · 2018 · Journal of Alzheimer s Disease
32. AAV- based vector improvements unrelated to capsid protein modification
    Ekaterina M. Shitik, Igor K. Shalik, Dmitry V. Yudkin · 2023 · Frontiers in Medicine
33. Host Cell Restriction Factors Blocking Efficient Vector Transduction: Challenges in Lentiviral and Adeno-Associated Vector Based Gene Therapies
    Ana Sofia Coroadinha · 2023 · Cells
34. Gene Therapy for Hemophilia: Progress to Date
    John C. Chapin, Paul Edward Monahan · 2017 · BioDrugs
35. Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy
    Purdy, Ryan, John, Molly, Bray, Alissa, Clare, Alison J., Copland, David A., Chan, Ying Kai, Henderson, Robert H., Nerinckx, Fanny, Leroy, Bart P., Yang, Paul et al. · 2025 · Progress in Retinal and Eye Research
36. Current limitations of gene therapy for rare pediatric diseases: Lessons learned from clinical experience with AAV vectors
    Antoine Gardin, Giuseppe Ronzitti · 2023 · Archives de Pédiatrie
37. Therapeutic application of the CRISPR system: current issues and new prospects
    Minyoung Lee, Seokjoong Kim · 2019 · Human Genetics
38. Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity
    Federico Mingozzi, Hildegard Büning · 2015 · Frontiers in Immunology
39. Covert Warfare Against the Immune System: Decoy Capsids, Stealth Genomes, and Suppressors
    Brad E. Hoffman, Roland W. Herzog · 2013 · Molecular Therapy
40. Recombinant adeno-associated virus vectors in the treatment of rare diseases
    Eric L. Hastie, Richard Jude Samulski · 2015 · Expert Opinion on Orphan Drugs
41. An innate immune response to adeno-associated virus genomes decreases cortical dendritic complexity and disrupts synaptic transmission
    Suriano, Christos M., Kumar, Neerav, Verpeut, Jessica L., Ma, Jie, Jung, Caroline, Dunn, Connor E., Carvajal, Brigett V., Nguyen, Ai Vy, Boulanger, Lisa M. · 2024 · Molecular Therapy
42. Retina-directed gene therapy: Achievements and remaining challenges
    Josef Biber, Catharina L.A. Gandor, Elvir Bećirović, Stylianos Michalakis · 2025 · Pharmacology & Therapeutics
43. Applications of adeno-associated virus vector-mediated gene delivery for neurodegenerative diseases and psychiatric diseases: Progress, advances, and challenges
    Amina Abulimiti, Michael Siu‐Lun Lai, Raymond Chuen‐Chung Chang · 2021 · Mechanisms of Ageing and Development
44. Preclinical models to assess the immunogenicity of AAV vectors
    Hildegund C.J. Ertl · 2017 · Cellular Immunology
45. Risk mitigation of immunogenicity: a key to personalized retinal gene therapy
    Varin, Juliette, Morival, Clément, Maillard, Noémien, Adjali, Oumeya, Cronin, Therese · 2021 · International Journal of Molecular Sciences
46. Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases
    Leyao Li, Lakshmy Vasan, Bryan Kartono, Kevan P. Clifford, Ahmadreza Attarpour, Raghav Sharma, Matthew Mandrozos, Ain Kim, Wenda Zhao, Ari Belotserkovsky et al. · 2023 · Biomedicines
47. Infusion reactions to adeno‐associated virus (AAV)‐based gene therapy: Mechanisms, diagnostics, treatment and review of the literature
    Kin Israel Notarte, Jesus Alfonso Catahay, Raymart Macasaet, Jin Liu, Jacqueline Veronica Velasco, Princess Juneire Peligro, Jolaine Vallo, Nathaniel Goldrich, Lokesh Lahoti, Jiayan Zhou et al. · 2023 · Journal of Medical Virology
48. Decoding cellular mechanism of recombinant adeno-associated virus (rAAV) and engineering host-cell factories toward intensified viral vector manufacturing
    Yongdan Wang, Qiang Fu, So Young Park, Yong Suk Lee, Seo‐Young Park, Dong‐Yup Lee, Seongkyu Yoon · 2024 · Biotechnology Advances
49. Gene Transfer of ZMapp Antibodies Mediated by Recombinant Adeno-Associated Virus Protects Against Ebola Infections
    Marc-André Robert, Nasha Nassoury, Parminder S. Chahal, Marie-Hélène Venne, Trina Racine, Xiangguo Qiu, Gary Kobinger, Amine Kamen, Rénald Gilbert, Bruno Gaillet · 2017 · Human Gene Therapy
50. Determinants of successful AAV-vectored delivery of HIV-1 bNAbs in early life
    Amir Ardeshir, Daniel O’Hagan, Isha D. Mehta, Siddhartha Shandilya, Lincoln Hopkins, Lourdes Adamson, Marcelo J. Kuroda, Patricia A. Hahn, Lucas A. B. da Costa, Sebastian P. Fuchs et al. · 2025 · Nature
51. Rational engineering of adeno‐associated virus capsid enhances human hepatocyte tropism and reduces immunogenicity
    Jiabao Han, Liyu Zhu, Jingwen Zhang, Lu Guo, Xuehan Sun, Cheng Huang, Kai Xu, Ying Zhang, Wei Li, Qi Zhou · 2022 · Cell Proliferation
52. Current and future prospects for hemophilia gene therapy
    Peter Ward, Christopher Walsh · 2016 · Expert Review of Hematology
53. Innate Immune Response Following AAV Administration
    Daniyar Dauletbekov, Julia K. Pfromm, A. K. Fritz, Manuel Dominik Fischer · 2019 · Advances in experimental medicine and biology
54. AAV Vectored Immunoprophylaxis for Filovirus Infections
    Amira D. Rghei, Laura P. van Lieshout, Lisa A. Santry, Matthew M. Guilleman, Sylvia P. Thomas, Leonardo Susta, Khalil Karimi, Byram W. Bridle, Sarah K. Wootton · 2020 · Tropical Medicine and Infectious Disease
55. Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary
    Jill A. Morris, Chris H. Boshoff, Nina Felice Schor, Ling M. Wong, Guangping Gao, Beverly L. Davidson · 2021 · Molecular Therapy
56. Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette
    Sandeep R. P. Kumar, Jun Xie, Shilang Hu, Jihye Ko, Qifeng Huang, Harrison C. Brown, Alok Srivastava, David M. Markusic, Christopher B. Doering, Harold Trent Spencer et al. · 2021 · Molecular Therapy — Methods & Clinical Development
57. New insights into the role of IFN-α/β and TLR7/8/9 in cancer immunotherapy and systemic autoimmunity
    Arthur Μ. Krieg · 2025 · Journal for ImmunoTherapy of Cancer
58. FSHD Therapeutic Strategies: What Will It Take to Get to Clinic?
    Charis L. Himeda, Peter L. Jones · 2022 · Journal of Personalized Medicine
59. Delivery challenges for CRISPR—Cas9 genome editing for Duchenne muscular dystrophy
    Made Harumi Padmaswari, Shilpi Agrawal, Mary S. Jia, Allie Ivy, Daniel A. Maxenberger, Landon A. Burcham, Christopher E. Nelson · 2023 · Biophysics Reviews
60. A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors
    Meredith E. Davis-Gardner, Jesse A. Weber, Jun Xie, Katja Pekrun, Eric A. Alexander, Kim L. Weisgrau, Jessica R. Furlott, Eva G. Rakasz, Mark A. Kay, Guangping Gao et al. · 2023 · Frontiers in Immunology
61. Developments in the treatment of hemophilia B: focus on emerging gene therapy
    John T. Gray, Maria Cancio, Ulrike M. Reiss, Amit Chunilal Nathwani, Andrew M. Davidoff · 2013 · The Application of Clinical Genetics
62. Alpha 1 Antitrypsin Gene Therapy Extends the Lifespan of Lupus-Prone Mice
    Ahmed Samir Elshikha, Ye Yuan, Yuanqing Lu, Mong‐Jen Chen, Georges Abboud, Mohammad Ahsanul Akbar, Henrike Plate, Hedwig Wolney, Tanja Hoffmann, Eleni Tagari et al. · 2018 · Molecular Therapy — Methods & Clinical Development
63. Viral and nonviral nanocarriers for in vivo CRISPR-based gene editing
    Zhongyuan Guo, Audrey T. Zhu, Ronnie H. Fang, Liangfang Zhang · 2024 · Nano Research
64. Successful AAV8 readministration: Suppression of capsid‐specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease
    Su Jin Choi, John S. Yi, Jeong‐A Lim, Thomas F. Tedder, Dwight D. Koeberl, William R. Jeck, Ankit K. Desai, Amy S. Rosenberg, Baodong Sun, Priya Sunil Kishnani · 2023 · The Journal of Gene Medicine
65. Tolerance and Cross-Tolerance following Toll-Like Receptor (TLR)-4 and -9 Activation Are Mediated by IRAK-M and Modulated by IL-7 in Murine Splenocytes
    MARK W. JULIAN, Heather R. Strange, Megan N. Ballinger, Richard S. Hotchkiss, Tracey L. Papenfuss, Elliott D. Crouser · 2015 · PLoS ONE
66. Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies
    Dongsheng Duan, Roland W. Herzog · 2026 · Molecular Therapy
67. Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
    Alexander Kistner, Jessica A. Chichester, Lili Wang, Roberto Calcedo, Jenny A. Greig, Leah N. Cardwell, Margaret C. Wright, Julien Couthouis, Sunjay Sethi, Brian E. McIntosh et al. · 2023 · Gene Therapy
68. Redundancy in Innate Immune Pathways That Promote CD8+ T-Cell Responses in AAV1 Muscle Gene Transfer
    Ning Li, Sandeep R. P. Kumar, Di Cao, Maite Muñoz-Melero, Sreevani Arisa, Bridget A. Brian, Calista M. Greenwood, Kentaro Yamada, Dongsheng Duan, Roland W. Herzog · 2024 · Viruses
69. Neuron-targeted gene therapy rescues multiple phenotypes of STXBP1-related disorders in mice and is well tolerated in nonhuman primates
    Rangoli Aeran, Annie Tanenhaus, Sheila M. S. Sears, Nathan J. Moerke, Adam Miller, Camille Artur, Yosr Bouhlal, Peter F. Bove, Alexander J. Diaz de Arce, Saki Shimizu et al. · 2025 · Molecular Therapy
70. AAV mediated gene therapy for haemophilia B: From the early attempts to modern trials
    Vincent Muczynski, Amit Chunilal Nathwani · 2024 · Thrombosis Research
71. A sensitive and drug tolerant assay for detecting anti-AAV9 antibodies using affinity capture elution
    Emma M. Simmons, Yi Wen, Jingling Li, Yuewei Qian, Li Chin Wong, Robert J. Konrad, Nicoletta Bivi · 2022 · Journal of Immunological Methods
72. Essential role of pre-existing humoral immunity in TLR9-mediated type I IFN response to recombinant AAV vectors in human whole blood
    Nada Salem Alakhras, Christopher A. Moreland, Li Chin Wong, Priyam Raut, Sid Kamalakaran, Yi Wen, Robert W. Siegel, Laurent P. Malherbe · 2024 · Frontiers in Immunology
73. rAAV expressing a COBRA-designed influenza hemagglutinin generates a protective and durable adaptive immune response with a single dose
    Kristin Wiggins, Stephen M. Winston, Isaiah L. Reeves, Jessica A Gaevert, Yunyu Spence, Mark A. Brimble, Brandi Livingston, Christopher L. Morton, Paul Glyndwr Thomas, Andrea J. Sant et al. · 2024 · Journal of Virology
74. Immunogenicity risk assessment of empty capsids present in adeno-associated viral vectors using predictive innate immune responses
    Nicole L. Jarvi, Kirk Hofman, Aditi Venkatesh, Emily Gorecki, Sathy V. Balu‐Iyer · 2024 · Journal of Pharmaceutical Sciences
75. Residual DNA impurities in AAV vectors—nature and transcription
    Hsin‐I Jen, Patrick F. Wilkinson, Xiaohui Lu, Wei Zhang · 2025 · Molecular Therapy — Methods & Clinical Development
76. Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate
    Prashant Kumar, Michael Wang, Ozan S. Kumru, John M. Hickey, Julio C. Sanmiguel, Nerea Zabaleta, Luk H. Vandenberghe, Sangeeta B. Joshi, David B. Volkin · 2023 · Molecular Therapy — Methods & Clinical Development
77. Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies
    Giovanni Di Minno, Wolfgang Miesbach, Giancarlo Castaman, Flora Peyvandi · 2024 · Haematologica
78. Novel Immune Response Evasion Strategy to Redose Adeno-associated Viral Vectors and Prolong Survival in Surfactant Protein-B–Deficient Mice
    Martin H. Kang, Sylvia P. Thomas, Caralyn Westley, Thomas Blouin, Liqun Xu, Ying Chan, Erin Lisk, Sarah Allen, Arul Vadivel, Kennedy Nangle et al. · 2025 · American Journal of Respiratory Cell and Molecular Biology
79. Immune responses in retinal gene therapy: challenges, mechanisms, and future strategies
    Yawei Ma, Yin Shen · 2025 · Frontiers in Immunology
80. Systemic delivery of AAV5, AAV8, and AAV9 packaging a C5-12-microdystrophin-FLAG expression cassette in non-human primates
    Mengping Liu, Erica Cook, Yan‐Shan Dai, Erich Ehlert, Francois du Plessis, Jacek Lubelski, Bogdan G. Sleczka, Petia A. Shipkova, Zhuyin Li, Joshua T. Gamse et al. · 2025 · Molecular Therapy — Methods & Clinical Development
81. Emerging Strategies of Cell and Gene Therapy Targeting Tumor Immune Microenvironment
    Runtian Wang, Xiaorui Duan, Jian Li, Cheng Zhang, Lin Shen · 2025 · Clinical Cancer Research
82. Preexisting Neutralizing Antibodies against Different Adeno-Associated Virus Serotypes in Humans and Large Animal Models for Gene Therapy
    Divya Ail, Deniz Dalkara · 2023 · Advances in experimental medicine and biology
83. Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies
    Steven W. Pipe, Allen Poma, Anita Rajasekhar, Tamara Everington, Serap Sankoh, Jack Allen, Jason Cataldo, Eric A. Crombez · 2025 · Blood Advances
84. Serotype-specific Binding Properties and Nanoparticle Characteristics Contribute to the Immunogenicity of rAAV1 Vectors
    Maxime Ferrand, Sylvie Da Rocha, Guillaume Corre, Anne H. M. Galy, Florence Boisgérault · 2015 · Molecular Therapy
85. The interplay of post-translational modification and gene therapy
    Victor Chukwudi Osamor, Shalom Nwodo Chinedu, Dominic E. Azuh, Emeka Iweala, Olubanke Olujoke Ogunlana · 2016 · Drug Design Development and Therapy
86. Adeno-Associated Virus Vectors—a Target of Cellular and Humoral Immunity—are Expanding Their Reach Toward Hematopoietic Stem Cell Modification and Immunotherapies
    Angela E. Araujo, Martin Bentler, Xabier Perez Garmendia, Asma Kaleem, Claire Fabian, Michael Alexander Morgan, Ulrich Thorsten Hacker, Hildegard Büning · 2024 · Human Gene Therapy
87. Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application
    Neel Mehta, Rénald Gilbert, Parminder S. Chahal, María Moreno, Nasha Nassoury, Nathalie Coulombe, Richard Gingras, Alaka Mullick, Simon Drouin, Marc Sasseville et al. · 2025 · Gene Therapy
88. “D” matters in recombinant AAV DNA packaging
    Junping Zhang, Ping Guo, Yinxia Xu, Patrick L. Mulcrone, Richard Jude Samulski, Weidong Xiao · 2021 · Molecular Therapy
89. The presence of CpGs in AAV gene therapy vectors induces a plasmacytoid dendritic cell-like population very early after administration
    Glenn, Justin D., Negash, Henos, Henry, William, Qian, Randolph, Liu, Ye, Danos, Olivier, Bruder, Joseph T., Karumuthil-Melethil, Subha · 2024 · Cellular Immunology
90. Long-term durability of rAAV gene therapy in hemophilia: Factor expression, clinical outcomes and underlying molecular mechanisms
    Yesim G. Dargaud, Gabriela G Yamaguti-Hayakawa, Jéssica O. Frade-Guanaes, Alexandre Leuci, Margareth C. Ozelo · 2025 · Blood Reviews
91. Factor IX Padua for haemophilia B gene addition: universal adaptation and repeated success
    Lindsey A. George · 2022 · The Lancet Haematology
92. Current regulatory requirements for assessment of immunogenicity for gene therapy medicinal products
    Christopher J. Mann, Jon Giblin, Manuela Braun, Felicitas Schmid, Maria Rathmann Sørensen, Paolo Caferra, Anett Hudák, Tamás Letoha, Núria Coderch, Timothy Paul Hickling et al. · 2025 · Cell Reports Medicine
93. Inhibition of immune response reduces pathology in dorsal root ganglia and peripheral nerves in cynomolgus macaques following AAV gene therapy
    Branka Grubor, Kate L. Henry, Su Jing Chan, Mark J. Sheehan, Anumeha Shah, Alex Pellerin, Judy Bai, Prasad Nadella, Santhosh Bommegowda, Patrick F. Cullen et al. · 2025 · Molecular Therapy — Methods & Clinical Development
94. Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization
    Tatiana S. Tsaregorodtseva, Maria A. Radyukhina, Aisylu I. Ayupova, Valeriya V. Solovyeva, Albert Akramovich Sufianov, Г. З. Суфианова, Albert Anatolyevich Rizvanov · 2026 · International Journal of Molecular Sciences
95. Safety Evaluation of Ocular Drugs
    Meg Ferrell Ramos, Mayssa Attar, Jennifer R. Seals, Keith A. Luhrs · 2024 · Elsevier eBooks
96. Pre-Existing Immunity to a Nucleic Acid Contaminant-Derived Antigen Mediates Transaminitis and Resultant Diminished Transgene Expression in a Mouse Model of Hepatic Recombinant Adeno-Associated Virus-Mediated Gene Transfer
    Mark A. Brimble, Christopher L. Morton, Stephen M. Winston, Isaiah L. Reeves, Yunyu Spence, Pei-Hsin Cheng, Junfang Zhou, Amit Chunilal Nathwani, Paul Glyndwr Thomas, Aisha Hegab Souquette et al. · 2024 · Human Gene Therapy
97. AAV Vector-Based Gene Therapy, Progress and Current Challenges
    Klaudia Kuranda, Federico Mingozzi · 2017 · Springer eBooks
98. Challenges in development and authorisation of gene therapy products
    Солдатов Александр Алексеевич, Ж. И. Авдеева, Горенков Дмитрий Витальевич, L. M. Khantimirova, S. G. Guseva, Вадим Анатольевич Меркулов · 2022 · Biological Products Prevention Diagnosis Treatment
99. Structural basis for Rep-mediated adeno-associated virus packaging.
    V. Xu, Aiysha McInnes, M. Wake, Marta Acebrón-García-de-Eulate, Joseph D Barritt, D. Bubeck, Sarah L. Rouse · 2026 · Cell Reports
100. Expanding the Potential of Gene Therapy for Duchenne Muscular Dystrophy
     Gyas Khan, Md Sadique Hussain · 2025 · Current Pharmaceutical Design