Anguela XM and High KA 2019 Entering the modern era of gene therapy. Annu. Rev. Med. 70 273–288

Article  CAS  PubMed  Google Scholar 

Appledorn DM, Patial Sonika, McBride Aaron, et al. 2008 Adenovirus vector-induced innate inflammatory mediators, MAPK signaling, as well as adaptive immune responses are dependent upon both TLR2 and TLR9 in vivo. J. Immunol. 181 2134–2144

Arabi F, Mansouri V and Ahmadbeigi N 2022 Gene therapy clinical trials, where do we go? An overview. Biomed. Pharmacother. 153 113324

Article  CAS  PubMed  Google Scholar 

Arber W and Linn S 1969 DNA modification and restriction. Annu. Rev. Biochem. 38 467–500

Article  CAS  PubMed  Google Scholar 

Asokan A, Conway JC, Phillips JL, et al. 2010 Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat. Biotechnol. 28 79–82

Article  CAS  PubMed  Google Scholar 

Ayuso E, Mingozzi F and Bosch F 2010 Production, purification and characterization of adeno-associated vectors. Curr. Gene Ther. 10 423–436

Article  CAS  PubMed  Google Scholar 

Bailey AM, Arcidiacono J, Benton KA, et al. 2015 United States Food and Drug Administration regulation of gene and cell therapies; in Regulatory aspects of gene therapy and cell therapy products (Eds.) MC Galli and M Serabian (Springer International Publishing) pp 1–29

Bennett A, Hull J, Jolinon N, et al. 2021 Comparative structural, biophysical, and receptor binding study of true type and wild type AAV2. J. Struct. Biol. 213 107795

Article  CAS  PubMed  PubMed Central  Google Scholar 

Benskey MJ, Kuhn NC, Galligan JJ, et al. 2015 Targeted gene delivery to the enteric nervous system using AAV: A comparison across serotypes and capsid mutants. Mol. Ther. 23 488–500

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bhattacharya A and Bhattacharya S 2019 Patient-driven initiatives for prioritizing drug discovery for rare diseases. Indian J. Med. Res. 149 326–328

Article  PubMed  PubMed Central  Google Scholar 

Bhattacharya S, Katoch VM and Majumder PP 2016 Current knowledge and new possibilities. Proc. Indian Nat. Sci. Acad. 82 1183–1187

Article  Google Scholar 

Blaese RM, Culver KW, Miller AD, et al. 1995 T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 270 475–480

Article  ADS  CAS  PubMed  Google Scholar 

Bostick B, Ghosh A, Yue Y, et al. 2007 Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration. Gene Ther. 14 1605–1609

Article  CAS  PubMed  Google Scholar 

Boycott KM, Lau LP, Cutillo CM, et al. 2019 International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseases. EMBO Mol. Med. 11 10486

Article  Google Scholar 

Büning H and Srivastava A 2019 Capsid modifications for targeting and improving the efficacy of AAV vectors. Mol. Ther. Methods Clin. Dev. 12 248–265

Article  PubMed  PubMed Central  Google Scholar 

Butera S, Tavarozzi R, Brunello L, et al. 2023 The black swan: a case of central nervous system graft-versus-host disease. J. Basic Clin. Physiol. Pharmacol. 34 805–809

Article  PubMed  Google Scholar 

Capasso C, Garofalo M, Hirvinen M, et al. 2014 The evolution of adenoviral vectors through genetic and chemical surface modifications. Viruses 6 832–855

Article  CAS  PubMed  PubMed Central  Google Scholar 

Chamberlain JS, Robb M, Braun S, et al. 2023 Microdystrophin expression as a surrogate endpoint for duchenne muscular dystrophy clinical trials. Hum. Gene Ther. 34 404–415

Article  CAS  PubMed  PubMed Central  Google Scholar 

Chand D, Mohr F, McMillan H, et al. 2021a Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy. J. Hepatol. 74 560–566

Article  CAS  PubMed  Google Scholar 

Chand DH, Zaidman C, Arya K, et al. 2021b Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: a case series. J. Pediatr. 231 265–268

Article  CAS  PubMed  Google Scholar 

Choudhury MC and Chaube P 2022 Integrating rare disease management in public health programs in India: exploring the potential of National Health Mission. Orphanet J. Rare Dis. 17 43

Article  PubMed  PubMed Central  Google Scholar 

Choudhury MC and Saberwal G 2019 The role of patient organizations in the rare disease ecosystem in India: an interview-based study. Orphanet J. Rare Dis. 14 1–14

Article  Google Scholar 

Cupelli K and Stehle T 2011 Viral attachment strategies: the many faces of adenoviruses. Curr. Opin. Virol. 1 84–91

Article  CAS  PubMed  Google Scholar 

Daniel HD, Kumar S, Kannangai R, et al. 2021 Prevalence of adeno-associated virus 3 capsid binding and neutralizing antibodies in healthy and hemophilia B individuals from India. Hum. Gene Ther. 32 451–457

Article  CAS  PubMed  Google Scholar 

Daniel HD, Kumar S, Kannangai R, et al. 2022 Age-stratified adeno-associated virus serotype 3 neutralizing and total antibody prevalence in hemophilia A patients from India. J. Med. Virol. 94 4542–4547

Article  CAS  PubMed  Google Scholar 

Daya S and Berns KI 2008 Gene therapy using adeno-associated virus vectors. Clin. Microbiol. Rev. 21 583–593

Article  CAS  PubMed  PubMed Central  Google Scholar 

Deverman BE, Ravina BM and Bankiewicz KS 2018 Gene therapy for neurological disorders: progress and prospects. Nat. Rev. Drug Discov. 17 641–659

Article  CAS  PubMed  Google Scholar 

Doering CB, Denning G and Shields JE 2018 Preclinical development of a hematopoietic stem and progenitor cell bioengineered factor VIII lentiviral vector gene therapy for hemophilia A. Hum. Gene Ther. 29 1183–1201

Article  CAS  PubMed  PubMed Central  Google Scholar 

Dong W and Kantor B 2021 Lentiviral vectors for delivery of gene-editing systems based on CRISPR/Cas: current state and perspectives. Viruses 13 1288

Article  CAS  PubMed  PubMed Central  Google Scholar 

Duan D, Goemans N, Takeda S, et al. 2021 Duchenne muscular dystrophy. Nat. Rev. Dis. Primers 7 13

Article  PubMed  PubMed Central  Google Scholar 

Dwivedi A, Karulkar A, Ghosh S, et al. 2019 Lymphocytes in cellular therapy: functional regulation of CAR T cells. Front. Immunol. 9 3180

Article  PubMed  PubMed Central  Google Scholar 

Edelstein ML, Abedi MR, Wixon J, et al. 2004 Gene therapy clinical trials worldwide 1989–2004 – an overview. J. Gene Med. 6 597–602

Article  PubMed  Google Scholar 

Edelstein ML, Abedi MR and Wixon J 2007 Gene therapy clinical trials worldwide to 2007- An update. J. Gene Med. 9 833–842

Article  PubMed  Google Scholar 

Elverum K and Whitman M 2020 Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine. Gene Ther. 27 537–544

Article  CAS  PubMed  Google Scholar 

Ertl HCJ 2022 Immunogenicity and toxicity of AAV gene therapy. Front. Immunol. 13 975803

Article  CAS  PubMed  PubMed Central  Google Scholar 

Farkas AM, Mariz S, Stoyanova-Beninska V, et al. 2017 Advanced therapy medicinal products for rare diseases: state of play of incentives supporting development in Europe. Front. Med. 16 53

Article  Google Scholar 

FDA 2015 Considerations for the design of early-phase clinical trials of cellular and gene therapy products (https://www.fda.gov/media/106369/download)

Florea M, Nicolaou F, Pacouret S, et al. 2023 High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography. Mol. Ther. Methods Clin. Dev. 28 146–159

Article  CAS  PubMed  Google Scholar 

Flotte TR and Berns KI 2005 Adeno-associated virus: a ubiquitous commensal of mammals. Hum. Gene Ther. 16 401–407

Article  CAS  PubMed  Google Scholar 

Frederiksen SD, Avramović V, Maroilley T, et al. 2022 Rare disorders have many faces: in silico characterization of rare disorder spectrum. Orphanet J. Rare Dis. 17 76

Article  PubMed  PubMed Central  Google Scholar 

Gao J, Mese K, Bunz O, et al. 2019 State-of-the-art human adenovirus vectorology for therapeutic approaches. FEBS Lett. 593 3609–3622