Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin
Abstract
Gene therapy for tuberous sclerosis type 2 proved beneficial in a mouse model of the disease, extending life span.
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- Jan 08, 2021
- Source ID
- 10.1126/sciadv.abb1703
Entities
People
- Anat Stemmer-Rachamimov
- Bakhos A Tannous
- Bence György
- Casey A Maguire
- David J Kwiatkowski
- David Yellen
- Elizabeth A. Thiele
- King Hwa Ling
- Masao Kaneki
- Pike See Cheah
- Roberta L. Beauchamp
- Roderick T. Bronson
- Shilpa Prabhakar
- Shingo Kasamatsu
- Vijaya Ramesh
- Xandra O. Breakefield
- Xuan Zhang
Organizations
- Harvard Medical School
- Massachusetts General Hospital
- National Institutes of Health
- Shriners Hospitals for Children
- United States Department of Defense
- University of Basel