Gene Therapy Fully Restores Vision to the All-Cone Nrl(−/−)Gucy2e(−/−) Mouse Model of Leber Congenital Amaurosis-1

Mutations in GUCY2D are the cause of Leber congenital amaurosis type 1 (LCA1). GUCY2D encodes retinal guanylate cyclase-1 (retGC1), a protein expressed exclusively in outer segments of photoreceptors and essential for timely recovery from photoexcitation. Recent clinical data show that, despite a hi...

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Foilsithe in:Hum Gene Ther
Main Authors: Boye, Sanford L., Peterson, James J., Choudhury, Shreyasi, Min, Seok Hong, Ruan, Qing, McCullough, K. Tyler, Zhang, Zhonghong, Olshevskaya, Elena V., Peshenko, Igor V., Hauswirth, William W., Ding, Xi-Qin, Dizhoor, Alexander M., Boye, Shannon E.
Formáid: Artigo
Teanga:Inglês
Foilsithe: Mary Ann Liebert, Inc. 2015
Ábhair:
Research Articles
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4575531/
https://ncbi.nlm.nih.gov/pubmed/26247368
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.053
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