Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

BACKGROUND: Choroideremia is an X-linked recessive disease that leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 (REP1). We assessed the effects of retinal gene therapy with an adeno-associated viral (AAV) vector encoding REP1 (AAV.REP1) in patients with th...

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Hlavní autoři: MacLaren, Robert E, Groppe, Markus, Barnard, Alun R, Cottriall, Charles L, Tolmachova, Tanya, Seymour, Len, Clark, K Reed, During, Matthew J, Cremers, Frans P M, Black, Graeme C M, Lotery, Andrew J, Downes, Susan M, Webster, Andrew R, Seabra, Miguel C
Médium: Artigo
Jazyk:Inglês
Vydáno: Lancet Publishing Group 2014
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4171740/
https://ncbi.nlm.nih.gov/pubmed/24439297
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S0140-6736(13)62117-0
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