26
- Chew WL, et al. A multifunctional AAV-CRISPR-Cas9 and its host response. Nat Methods.
2016;13:868–74. - Tabebordbar M, et al. In vivo gene editing in dystrophic mouse muscle and muscle stem cells.
Science. 2016;351:407–11. - Hung SSC, et al. AAV-mediated CRISPR/Cas gene editing of retinal cells in vivo. Invest
Opthalmol Vis Sci. 2016;57:3470. - Yang Y, et al. A dual AAV system enables the Cas9-mediated correction of a metabolic liver
disease in newborn mice. Nat Biotechnol. 2016;34:334–8. - Brockstedt DG, et al. Induction of immunity to antigens expressed by recombinant adeno-
associated virus depends on the route of administration. Clin Immunol. 1999;92:67–75. - Mingozzi F, High KA. Immune responses to AAV in clinical trials. Curr Gene Ther.
2011;11:321–30. - Rogers GL, et al. Innate immune responses to AAV vectors. Front Microbiol. 2011; 2.
- Denard J, et al. Human galectin 3 binding protein interacts with recombinant adeno- associated
virus type 6. J Virol. 2012;86:6620–31. - Li C, et al. Development of patient-specific AAV vectors after neutralizing antibody selection
for enhanced muscle gene transfer. Mol Ther. 2016;24:53–65. - Tseng Y-S, Agbandje-McKenna M. Mapping the AAV capsid host antibody response toward
the development of second generation gene delivery vectors. Front Immunol. 2014;5:9. - Li S, et al. Efficient and targeted transduction of nonhuman primate liver with systemically
delivered optimized AAV3B vectors. Mol Ther. 2015;23:1867–76. - Murlidharan G, Corriher T, Ghashghaei HT, Asokan A. Unique glycan signatures regulate
adeno-associated virus tropism in the developing brain. J Virol. 2015;89:3976–87. - Castle M, Turunen H, Vandenberghe L, Wolfe J. Controlling AAV tropism in the nervous
system with natural and engineered capsids. In: Manfredsson FP, editor. Gene therapy for
neurological disorders. New York: Springer; 2016. p. 133–49. - Muzyczka N, Berns KI. AAV’s golden jubilee. Mol Ther. 2015;23:807–8.
- Flotte T, et al. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF
patients with mild lung disease. Hum Gene Ther. 1996;7:1145–59. - Gene Therapy Clinical Trials Worldwide. http://www.wiley.com/legacy/wileychi/genmed/
clinical/. Accessed 28 Jan 2017. - Nathwani AC, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia
B. N Engl J Med. 2014;371:1994–2004. - Bainbridge JWB, et al. Long-term effect of gene therapy on Leber’s congenital amaurosis. N
Engl J Med. 2015;372:1887–97. - Gaudet D, et al. Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X)
gene therapy for lipoprotein lipase deficiency: an open-label trial. Gene Ther. 2013;20:361–9. - Morrison C. $1-million price tag set for Glybera gene therapy. Nat Biotechnol. 2015;33:217–8.
- Moran N. First gene therapy approved. Nat Biotechnol. 2012;30:1153.
- Vercauteren K, et al. Superior in vivo transduction of human hepatocytes using engineered
AAV3 capsid. Mol Ther. 2016;24:1042–9. - Riddell SR, et al. Restoration of viral immunity in immunodeficient humans by the adoptive
transfer of T cell clones. Science. 1992;257:238–41. - Levine BL, et al. Adoptive transfer of costimulated CD4+ T cells induces expansion of
peripheral T cells and decreased CCR5 expression in HIV infection. Nat Med. 2002;8:47–53. - Rapoport AP, et al. Restoration of immunity in lymphopenic individuals with cancer by vac-
cination and adoptive T-cell transfer. Nat Med. 2005;11:1230–7. - Hoos A. Development of immuno-oncology drugs—from CTLA4 to PD1 to the next genera-
tions. Nat Rev Drug Discov. 2016;15:235–47. - June CH, Riddell SR, Schumacher TN. Adoptive cellular therapy: a race to the finish line. Sci
Transl Med. 2015;7:280ps7. - Irving BA, Weiss A. The cytoplasmic domain of the T cell receptor zeta chain is sufficient to
couple to receptor-associated signal transduction pathways. Cell. 1991;64:891–901.
J.E. DiCarlo et al.