New Rochelle, NY, Oct.5, 2017 -- A new study demonstrates success in using a shortened form of the CEP290 gene for gene therapy in a mouse model of Leber congenital amaurosis type 10 , a retinal degenerative disorder that causes childhood blindness. The large size of the full-length gene has proven difficult to package into the adeno-associated virus delivery vectors commonly used in gene therapy, but use of the truncated gene led to significant improvement in photoreceptor survival, morphology, and function, as reported in an article published in Human Gene Therapy , a peer-reviewed journal from Mary Ann Liebert, Inc., publishers .