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

Hemant Khanna, PhD and colleagues have published a study in the journal Human Gene Therapy (http://online.liebertpub.com/doi/abs/10.1089/hum.2017.049) 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. https://www.umassmed.edu/news/news-archives/2017/08/hemant-khanna-exploring-innovative-gene-therapy-for-severe-inherited-eye-disorder/