Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis

Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis

Mutations in GUCY2D are associated with severe early-onset retinal dystrophy, Leber congenital amaurosis type 1 (LCA1), a leading cause of blindness in children. Despite a high degree of visual disturbance stemming from photoreceptor dysfunction, patients with LCA1 largely retain normal photorecepto...

Full description

Bibliographic Details
Main Authors: Sanford L. Boye, Catherine O’Riordan, James Morris, Michael Lukason, David Compton, Rena Baek, Dana M. Elmore, James.J. Peterson, Diego Fajardo, K. Tyler McCullough, Abraham Scaria, Alison McVie-Wylie, Shannon E. Boye
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
adeno-associated virus
GUCY2D
Leber congenital amaurosis
LCA
LCA1
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001802

Internet

http://www.sciencedirect.com/science/article/pii/S2329050122001802

Similar Items