11 November 2015
Vol 7, Issue 313

About The Cover

Cover image expansion

ONLINE COVER Gene Therapy Lights Up. Shown is expression of recombinant enzyme (pseudo-colored blue) in the hippocampus of a dog with Batten disease after a single gene therapy treatment. In this naturally occurring dog model of a lysosomal storage disease, deficiency of the lysosomal enzyme TPP1 results in motor abnormalities, cognitive deficits, seizures, and vision loss. One-time delivery of the gene encoding the canine form of TPP1 (via an adeno-associated viral vector) to the cerebral ventricles of this dog model resulted in delayed disease progression, protection from cognitive decline and extended life span. (Katz et al.). [CREDIT: L. TECEDOR AND B. DAVIDSON/THE CHILDREN'S HOSPITAL OF PHILADELPHIA]