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Clinical In Vivo Gene Therapy Publications
Below is a list of selected publications from Clinical In Vivo Gene Therapy.
George LA. No CpGs for AAV?. Blood. 2021 Feb; 137(6):721-723. PMID: 33570612
Konkle B, Pierce G, Coffin D, Naccache M, Clark RC, George L, Iorio A, O'Mahony B, Pipe S, Skinner M, Watson C, Peyvandi F, Mahlangu J, ISTH subcommittee on Factor VIII, Factor IX, and Rare Bleeding Disorders. Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH. J Thromb Haemost. 2020 Nov; 18(11):3074-3077. PMID: 33463024
George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BS, Ozela M, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA. Long-Term Follow-Up of the First In Human Intravascular Delivery of AAV For Gene Transfer: AAV2 hFIX16 For Severe Hemophilia B. Mol Ther. 2020 Sep; 28(9):2073-2082. PMID: 32559433
Maguire AM, Russell S, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, Marshall KA, McCague S, Reichert H, Davis M, Simonelli F, Leroy BP, Wright JF, High KA, Bennett J.. Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials. Ophthalmology. 2019 Sep; 126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22. PMID: 31443789.
George LA, Sullivan SK, Giermasz G, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hu Di, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. Hemophilia B Gene Therapy with a High Specific Activity Factor IX Variant. N Engl J Med. 2017 Dec; 377(2):2215-2227. PMID: 29211678
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017 Aug; 390(10097):849-860 PMID: 28712537 Erratum in: Lancet. 2017 Aug 26;390(10097):848. PMID: 28850042
Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, Auricchio A, Pierce EA, Ruggiero J, Leroy BP, Simonelli F, High KA, Maguire AM. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Lancet. 2016 Aug; 388(10045):661-672. PMID: 27375040
Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, Simonelli F, Shindler KS, Mingozzi F, High KA, Maguire AM. AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med. 2012 Feb; 4(120):120ra15. PMID: 22323828
Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JIW, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, McDonnell JW, Leroy BP, Simonelli F, Bennett J. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet. 2009 Nov; 374(9701):1597-1605. PMID: 19854499. Erratum in: Lancet. 2010 Jan 2;375(9708):30
Maguire AM, Simonelli F, Pierce EA, Pugh EN, Jr., Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med. 2008 Apr; 358(21): 2240-8. PMID: 18441370
Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JEJ, Ragni MV, Manno CS, Sommer J, Jiang H, Pierce GF, Ertle HCJ, High KA. CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med. 2007 Apr; 13(4):419-422. PMID: 17369837
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nkai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med. 2006 Mar; 12(3):342-347. PMID: 16474400
Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DBG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood. 2003 Apr; 101(8):2963-2972