AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
Top Cited Papers
Open Access
- 3 September 2009
- journal article
- clinical trial
- Published by American Society of Hematology in Blood
- Vol. 114 (10) , 2077-2086
- https://doi.org/10.1182/blood-2008-07-167510
Abstract
In a clinical trial for adeno-associated virus serotype 1 (AAV-1)–mediated gene transfer to muscle for lipoprotein lipase (LPL) deficiency, 1 subjectKeywords
This publication has 42 references indexed in Scilit:
- Limb‐girdle muscular dystrophy type 2D gene therapy restores α‐sarcoglycan and associated proteinsAnnals of Neurology, 2009
- Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector–mediated gene therapyBlood, 2009
- Safety and Efficacy of Gene Transfer for Leber's Congenital AmaurosisNew England Journal of Medicine, 2008
- Effect of Gene Therapy on Visual Function in Leber's Congenital AmaurosisNew England Journal of Medicine, 2008
- Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liverBlood, 2007
- Initial T Cell Receptor Transgenic Cell Precursor Frequency Dictates Critical Aspects of the CD8+ T Cell Response to InfectionImmunity, 2007
- Acquisition of direct antiviral effector functions by CMV-specific CD4+ T lymphocytes with cellular maturationThe Journal of Experimental Medicine, 2006
- Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapyBlood, 2006
- Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primatesBlood, 2006
- Influence of Vector Dose on Factor IX-Specific T and B Cell Responses in Muscle-Directed Gene TherapyHuman Gene Therapy, 2002