Lentiviral vectors containing a retinal pigment epithelium specific promoter for leber congenital amaurosis gene therapy. Lentiviral gene therapy for LCA.
Bemelmans
Alexis-Pierre
AP
Oculogenetics Unit, Hôpital Ophtalmique Jules Gonin, Lausanne, Switzerland.
Kostic
Corinne
C
Hornfeld
Dana
D
Jaquet
Muriel
M
Crippa
Sylvain V
SV
Hauswirth
William W
WW
Lem
Janis
J
Wang
Zhongyan
Z
Schorderet
Daniel E
DE
Munier
Francis L
FL
Wenzel
Andreas
A
Arsenijevic
Yvan
Y
D000818:Animals; D002352:Carrier Proteins / Q000235:genetics; D004596:Electroretinography / Q000379:methods; D005136:Eye Proteins / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D049452:Green Fluorescent Proteins / Q000378:metabolism; D006801:Humans; D016086:Lentivirus / Q000235:genetics* / Q000378:metabolism; D051379:Mice; D018345:Mice, Knockout; D029242:Optic Atrophy, Hereditary, Leber / Q000235:genetics* / Q000628:therapy*; D010857:Pigment Epithelium of Eye / Q000378:metabolism*; D011401:Promoter Regions, Genetic; D012160:Retina / Q000378:metabolism; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase
D002352:Carrier Proteins; D005136:Eye Proteins; D049452:Green Fluorescent Proteins; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase