Popular on Patch: Lions Club Fundraising to Give Sight to Highlands Siblings
Brother and sister Patrick and Mirielle St. Arnaud have a rare congenital blindness disease, but, with proper funding, a new type of gene therapy could cure them.
Brother and sister Patrick and Mirielle St. Arnaud have a rare congenital blindness disease, but, with proper funding, a new type of gene therapy could cure them.
Brother and sister Patrick and Mirielle St. Arnaud have a rare congenital blindness disease, but, with proper funding, a new type of gene therapy could cure them.
When Patrick and Mirielle St. Arnaud were born blind from the eye disease Leber’s congenital amaurosis (LCA), it seemed unlikely that either would ever gain better vision. But a 2009 gene-therapy breakthrough has given hope, and more. Researchers at the Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine have begun successfully treating LCA with injections of healthy versions of children’s defective genes, effectively reversing the disease. However, LCA can be caused by defects in several different genes—and the one that affects the St. Arnaud siblings (NPHP5) has not yet been copied, a process that requires significant time and funding. Enter the La Grange Lions Club volunteer organization, who…
Darren McRoy
10:37 pm on Monday, December 10, 2012
Thank you for this great comment, Betsy!! :-)   more ›