Affiliation:
1. Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, California 92037
Abstract
ABSTRACT
Retinitis pigmentosa (RP) is the most common inherited retinal disease, in which photoreceptor cells degenerate, leading to blindness. Mutations in the rod photoreceptor cGMP phosphodiesterase β subunit (PDEβ) gene are found in patients with autosomal recessive RP as well as in the
rd
mouse. We have recently shown that lentivirus vectors based on human immunodeficiency virus (HIV) type 1 achieve stable and efficient gene transfer into retinal cells. In this study, we evaluated the potential of HIV vector-mediated gene therapy for RP in the
rd
mouse. HIV vectors containing a gene encoding a hemagglutinin (HA)-tagged PDEβ were injected into the subretinal spaces of newborn
rd
mouse eyes. One to three rows of photoreceptor nuclei were observed in the eyes for at least 24 weeks postinjection, whereas no photoreceptor cells remained in the eyes of control animals at 6 weeks postinjection. Expression of HA-tagged PDEβ in the rescued photoreceptor cells was confirmed by two-color confocal immunofluorescence analysis using anti-HA and anti-opsin antibodies. HIV vector-mediated gene therapy appears to be a promising means for the treatment of recessive forms of inherited retinal degeneration.
Publisher
American Society for Microbiology
Subject
Virology,Insect Science,Immunology,Microbiology
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