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Article

Gene therapy for eye as regenerative medicine? Lessons from RPE65 gene therapy for Leber's Congenital Amaurosis.

The international journal of biochemistry & cell biology - 1 Nov 2014

Rakoczy Elizabeth P, Narfström Kristina

Abstract excerpt

Recombinant virus mediated gene therapy of Leber's Congenital Amaurosis has provided a wide range of data on the utility of gene replacement therapy for recessive diseases. Studies to date demonstrate that gene therapy in the eye is safe and can result in long-term recovery of visual function, but they also highlight that further research is required to identify optimum intervention time-points, target...

Topics

Animals
Clinical Trials as Topic
Dependovirus
Genetic Therapy
Genetic Vectors
Humans
Leber Congenital Amaurosis
Mutation
Recombination, Genetic
Regenerative Medicine
Time Factors

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Treatment Outcome

Vision, Ocular

cis-trans-Isomerases

Source

Identifiers

PMID: 25286304
DOI: 10.1016/j.biocel.2014.09.022

Genes

RPE65

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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