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Study shows potential of CEP290 minigenes as therapeutics for childhood blindness disorder

Date Posted: Saturday, September 16, 2017

Hemant Khanna, PhD and colleagues have published a study in the journal Human Gene Therapy ( describing the use of minigenes as a therapeutic strategy for CEP290-associated Leber congenital amaurosis (LCA).

This strategy bypasses the roadblock posed by the large size of the full CEP290 gene, which is not compatible with the clinically approved adeno-associated viral (AAV) vector-mediated gene delivery.

Dr. Khanna image