Gene therapy restores vision in a canine model of childhood blindness

By G. Acland, G. Aguirre, J. Ray, Qi Zhang, T. Aleman, A. Cideciyan, S. Pearce-Kelling, V. Anand, Yong Zeng, A. Maguire and 3 more. Nature Genetics, 2001

1,227 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/ee336a03f83c8b09ae2cf0371baecc5d86a33776

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

1. S. Jacobson
2. A. Cideciyan
3. R. Ratnakaram
4. E. Héon
5. S. Schwartz
6. Matthew L. Batten
7. R. Calcedo
8. A. J. Roman

Citations in the corpus

• Gene Therapy for Leber Congenital Amaurosis caused by RPE65 mutations: Safety and Efficacy in Fifteen Children and Adults Followed up to Three Years (2011, background): “In a canine model of RPE65 deficiency, subretinal delivery of a gene-viral vector agent led to a remarkable activation of retinal and postretinal function.(4,5) Mice”
• Pharmacological and rAAV Gene Therapy Rescue of Visual Functions in a Blind Mouse Model of Leber Congenital Amaurosis (2005, positive): “A recombinant adenoassociated virus (rAAV) carrying wild-type (WT) Rpe65 (rAAV-Rpe65) restored visual function in this model of childhood blindness [9].”
• Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics (2008, background): “We used adeno-associated virus-2-based RPE65 gene replacement therapy to treat three young adults with RPE65-LCA and measured their vision before and up to 90 days after the intervention.”