In the coming years, CRISPR will democratize therapeutic genome editing for n=1 genetic diseases. These efforts will be sponsored by academic institutions and nonprofits, as opposed to companies, because they won’t be profitable.
n=1 genetic diseases: Ones that are so rare that there is likely only one, or up to a handful, cases diagnosed across the globe.
Why democratizing genome editing is important: Since developing a treatment or cure for this kind of diseases only benefits so few people, there isn’t enough financial incentive for companies. To help the often devastated patients, it’s up to the governments, academic institutions, and nonprofits to shoulder the cost of the development of these highly specific therapies.
Inspired by Dr. Fyodor Urnov’s keynote presentation at World CRISPR Day 2021. The recording can be found here: