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Comment
. 2013 May 7;110(19):E1706.
doi: 10.1073/pnas.1304296110.

Reply to Townes-Anderson: RPE65 gene therapy does not alter the natural history of retinal degeneration

Artur V Cideciyan, Samuel G Jacobson, William A Beltran, William W Hauswirth, Gustavo D Aguirre
Comment

Reply to Townes-Anderson: RPE65 gene therapy does not alter the natural history of retinal degeneration

Artur V Cideciyan et al. Proc Natl Acad Sci U S A. .
No abstract available

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Conflict of interest statement

Conflict of interest statement: W.W.H. and the University of Florida have a financial interest in the use of adeno-associated virus therapies, and own equity in a company (AGTC Inc.) that might, in the future, commercialize some aspects of this work. The University of Pennsylvania, University of Florida, and Cornell University hold a patent on the described gene-therapy technology (United States Patent 20070077228, “Method for Treating or Retarding the Development of Blindness”).

Comment on

References

    1. Townes-Anderson E. Increased levels of gene therapy may not be beneficial in retinal disease. Proc Natl Acad Sci USA. 2013;110:E1705. - PMC - PubMed
    1. Cideciyan AV, et al. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci USA. 2013;110(6):E517–E525. - PMC - PubMed
    1. Alfinito PD, Townes-Anderson E. Activation of mislocalized opsin kills rod cells: A novel mechanism for rod cell death in retinal disease. Proc Natl Acad Sci USA. 2002;99(8):5655–5660. - PMC - PubMed
    1. Beltran WA, et al. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Proc Natl Acad Sci USA. 2012;109(6):2132–2137. - PMC - PubMed
    1. Cideciyan AV, et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci USA. 2008;105(39):15112–15117. - PMC - PubMed

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