AAV vectors with myelin protein zero promoter and uses thereof for treating SCHWANN cell-associated diseases like CHARCOT-MARIE-TOOTH disease
Grant
US12582725B2
Kind: B2
Mar 24, 2026
Assignee
THE CYPRUS FOUNDATION FOR MUSCULAR DYSTROPHY RESEARCH
Inventors
Kleopas Kleopa, Alexia Kagiava, Natasa Schiza, Irene Sargiannidou
Abstract
The present invention provides viral vectors for use in the treatment and prevention of diseases associated with Schwann cells by delivering polynucleotides specifically to Schwann cells and achieving Schwann cell specific expression. The present invention has particular application in treatment and prevention of Charcot-Marie-Tooth disease and other demyelinating neuropathies. The preferred vectors are adeno-associated vectors (AAV) having a Schwann cell-specific promoter from the Myelin Protein Zero (Mpz, P0) or a minimal Mpz promoter.
CPC Classifications
C12N 15/86
C12N 2830/008
C12N 2330/51
C12N 2750/14143
A61P 25/00
A61P 21/00
A61P 25/02
A01K 2267/0318
C07K 14/705
Filing Date
2020-06-03
Application No.
17616006
Claims
10