Gene Therapy for Rare Diseases

Gene Therapy for Rare Diseases is organized by The New York Academy of Sciences and would be held on Apr 11, 2017 at The New York Academy Of Sciences, New York, United States of America.

Description
Numerous life-threatening rare diseases are due to single gene mutations that manifest within several months of birth. With no effective treatment options, these monogenic diseases require constant and expensive care. Gene therapy is beginning to show clinical promise as a strategy for treating rare diseases, but faces challenges around safe, consistent, and durable gene delivery to targeted tissues. This symposium will explore recent advances in gene therapy for rare diseases, identify issues that remain to be addressed in order for gene therapy to become more widely used, and explore opportunities for application to other disease areas.