Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?

Luiza Amara Maciel BragaCarlos Gilbert Conte FilhoFabio Batista Mota

Published in: Therapeutic advances in rare disease (2022)

Overall, the researchers who participated in this study expect that in the future genetic therapies will greatly benefit the treatment of patients with rare genetic diseases.

Keyphrases

genome wide
copy number
current status
replacement therapy