Document Detail

Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosis.
MedLine Citation:
PMID:  21299439     Owner:  NLM     Status:  MEDLINE    
INTRODUCTION: The positive results of pioneering clinical trials using gene therapy as treatment for patients with Leber congenital amaurosis (LCA) have ushered in a new era of molecular retinal therapeutics for LCA, other blinding retinal disorders and gene therapy applications.
AREAS COVERED: This review describes the role of retinal pigment epithelium-specific 65 kDa protein (RPE65) in the visual cycle and how RPE65 deficiency results in LCA; the extensive preclinical studies with recombinant adeno-associated virus (rAAV)-RPE65 gene vectors; and the human rAAV-RPE65 and related gene therapy clinical trials and studies. The literature search included a review of primary sources (e.g., journal articles) that reported study data results and key secondary sources such as meta-reviews available through PubMed, as well as reviews of clinical trial descriptions and results as reported in, conference publications and news releases.
EXPERT OPINION: LCA-RPE65 gene therapy is an example of successful, innovative, translational research. Further research is needed regarding how retinal gene therapy can be improved.
Linda Stein; Kamolika Roy; Lei Lei; Shalesh Kaushal
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Publication Detail:
Type:  Journal Article; Review    
Journal Detail:
Title:  Expert opinion on biological therapy     Volume:  11     ISSN:  1744-7682     ISO Abbreviation:  Expert Opin Biol Ther     Publication Date:  2011 Mar 
Date Detail:
Created Date:  2011-02-08     Completed Date:  2011-05-23     Revised Date:  2013-11-06    
Medline Journal Info:
Nlm Unique ID:  101125414     Medline TA:  Expert Opin Biol Ther     Country:  England    
Other Details:
Languages:  eng     Pagination:  429-39     Citation Subset:  IM    
University of Massachusetts Medical School, UMass Memorial Eye Center, Department of Ophthalmology, 281 Lincoln St., Worcester, MA 01605, USA.
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MeSH Terms
Carrier Proteins / genetics*,  metabolism
Dependovirus / genetics
Evidence-Based Medicine
Eye Proteins / genetics*,  metabolism
Gene Transfer Techniques
Genetic Therapy* / adverse effects
Genetic Vectors
Leber Congenital Amaurosis / genetics,  metabolism,  physiopathology,  therapy*
Retina / metabolism*,  physiopathology
Translational Medical Research
Treatment Outcome
Vision, Ocular
Reg. No./Substance:
0/Carrier Proteins; 0/Eye Proteins; EC isomerohydrolase; EC 5.2.-/cis-trans-Isomerases

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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