The newest addition to the NORD Rare Disease Video Library is a set of four videos on genome editing, a promising approach to treatment for many people affected by rare diseases. The videos address questions shared with NORD by our members and social media followers.
These short, animated videos answer basic questions such as “What is genome editing?” and “Can genome editing cure rare diseases?” They also address more general questions such as how to find out about clinical trials, how patient organizations can encourage genome editing research on their diseases, and how potential safety and ethical concerns are being addressed.
NORD developed the content based on the questions submitted to us by patients, caregivers, and leaders of patient organizations across a broad spectrum of rare diseases. The videos complement an earlier one on gene therapy that also addresses patient/caregiver questions. The gene therapy video is one of the most-viewed in NORD’s Rare Disease Video Library and has been translated into other languages at the request of patient organizations.
Philip John (P.J.) Brooks, PhD, and Deanna Portero of the NIH National Center for Advancing Translational Sciences served as medical reviewers for the genome editing videos to ensure the accuracy of the information.
The videos were made possible by a donation from Editas Medicine. NORD is solely responsible for the content.