The American Museum of Natural History's Genomic Revolution Exhibit features Canavan Gene Therapy
(May 26, 2001-January 1, 2002)
RECENT PRESS
Thanks to the groundbreaking research pioneered through your hard work, the latest study shows gene therapy stabilized eighteen children with Canavan Disease, completely stopping brain atrophy. For coverage on the study and for more information on gene therapy, see the following articles:
"Long Term Follow Up After Gene Therapy for Canavan Disease"
Science Journal of Translational Medicine
"A New Gene Therapy: An Old Hard TruthNational Geographic
"Canavan Disease"
CheckOrphan
“After years of frustration, in 2001 a clinical trial began to test the safety of inserting a therapeutic gene into the brains of 13 children with Canavan disease, including Lindsay.”
“Beating the Odds.” The Forward, December 23, 2019