Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis | Zendy

James Bainbridge | Zendy; Manjit Mehat | Zendy; Venki Sundaram | Zendy; Scott Robbie | Zendy; Susie E. Barker | Zendy; Caterina Ripamonti | Zendy; Anastasios Georgiadis | Zendy; Freya M. Mowat | Zendy; Stuart G. Beattie | Zendy; Peter J. Gardner | Zendy; Kecia L. Feathers | Zendy; Vy Luong | Zendy; Suzanne Yzer | Zendy; Kamaljit S. Balaggan | Zendy; Ananth C. Viswanathan | Zendy; Thomy de Ravel | Zendy; Ingele Casteels | Zendy; Graham E. Holder | Zendy; Nick Tyler | Zendy; Fred W. Fitzke | Zendy; Richard G. Weleber | Zendy; Marko Nardini | Zendy; Anthony T. Moore | Zendy; Debra A. Thompson | Zendy; Simon M. PetersenJones | Zendy; Michel Michaelides | Zendy; L. Ingeborgh van den Born | Zendy; Andrew Stockman | Zendy; Alexander J. Smith | Zendy; Gary S. Rubin | Zendy; Robin R. Ali | Zendy

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Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

Author(s) -

James Bainbridge,

Manjit Mehat,

Venki Sundaram,

Scott Robbie,

Susie E. Barker,

Caterina Ripamonti,

Anastasios Georgiadis,

Freya M. Mowat,

Stuart G. Beattie,

Peter J. Gardner,

Kecia L. Feathers,

Vy Luong,

Suzanne Yzer,

Kamaljit S. Balaggan,

Ananth C. Viswanathan,

Thomy de Ravel,

Ingele Casteels,

Graham E. Holder,

Nick Tyler,

Fred W. Fitzke,

Richard G. Weleber,

Marko Nardini,

Anthony T. Moore,

Debra A. Thompson,

Simon M. PetersenJones,

Michel Michaelides,

L. Ingeborgh van den Born,

Andrew Stockman,

Alexander J. Smith,

Gary S. Rubin,

Robin R. Ali

Publication year - 2015

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1414221

Subject(s) - rpe65 , medicine , genetic enhancement , retinal , electroretinography , ophthalmology , visual acuity , erg , cis trans isomerases , retina , retinal degeneration , retinal pigment epithelium , gene , neuroscience , biology , genetics , peptidylprolyl isomerase , isomerase

Mutations in RPE65 cause Leber's congenital amaurosis, a progressive retinal degenerative disease that severely impairs sight in children. Gene therapy can result in modest improvements in night vision, but knowledge of its efficacy in humans is limited.

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