Clinical In Vivo Gene Therapy Publications

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Below is a list of selected publications from Clinical In Vivo Gene Therapy.

No CpGs for AAV?
Blood · February, 2021
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 · November, 2020
Long-Term Follow-Up of the First In Human Intravascular Delivery of AAV For Gene Transfer: AAV2 hFIX16 For Severe Hemophilia B
Mol Ther · September, 2020
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials
Ophthalmology · September, 2019
Hemophilia B Gene Therapy with a High Specific Activity Factor IX Variant
N Engl J Med · December, 2017
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 · August, 2017
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 · August, 2016
AAV2 gene therapy readministration in three adults with congenital blindness
Sci Transl Med · February, 2012
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
Lancet · November, 2009
Safety and efficacy of gene transfer for Leber's congenital amaurosis
N Engl J Med · April, 2008
CD8(+) T-cell responses to adeno-associated virus capsid in humans
Nat Med · April, 2007
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
Nat Med · March, 2006
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
Blood · April, 2003