Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis-Reference-Cited by-同舟云学术

Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis

Published:2021-08 Issue:8 Volume:29 Page:2456-2468
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Aguirre Gustavo D.^ORCID,Cideciyan Artur V.,Dufour Valérie L.,Ripolles-García Ana,Sudharsan Raghavi,Swider Malgorzata,Nikonov Roman,Iwabe Simone,Boye Sanford L.,Hauswirth William W.,Jacobson Samuel G.,Beltran William A.

Funder

NIH

Foundation Fighting Blindness Inc

Research to Prevent Blindness

Hope for Vision

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

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5. Mainzer-Saldino syndrome is a ciliopathy caused by IFT140 mutations;Perrault;Am. J. Hum. Genet.,2012

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