Journal
VISION RESEARCH
Volume 43, Issue 8, Pages 919-926Publisher
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/S0042-6989(02)00389-9
Keywords
gene therapy; retinal disease; retinal degeneration; mutant; cell culture
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Funding
- NEI NIH HHS [R01 EY10820, EY 12156] Funding Source: Medline
- NIDDK NIH HHS [P30 DK 47757-05] Funding Source: Medline
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Choroideremia (CHM) is an X-linked retinal degenerative disease resulting from a lack of functional Rab Escort Protein-1 (REP-1). As a first step in developing gene-based therapies for this disease, we evaluated the feasibility of delivering functional REP-1 to defective lymphocytes and fibroblasts isolated from individuals with CHM. A recombinant adenovirus delivering the full-length human cDNA encoding REP-1 under the control of a cytomegalovirus promoter was generated. Adenovirus-mediated delivery of REP-1 rescued the defective cells as assessed through protein and enzymatic assays. Ultimately, it may be possible to use virus-mediated delivery of REP-1 to evaluate disease intervention in vivo. (C) 2002 Elsevier Science Ltd. All rights reserved.
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