A novel muscle-specific promoter
AssigneesGenethon, Inserm (Institut National De La Santé Et De La Recherch MéDicale), Universite D'Evry Val D'Essonne
InventorsWilliam LOSTAL, Isabelle RICHARD
Life of this patent
Estimating the term…
Abstract
The present invention concerns a novel short promoter characterized by a high activity in the skeletal muscles and a low activity in the heart. It then constitutes a valuable candidate especially for driving the expression of transgenes encoding proteins useful for the treatment of muscular dystrophies.
Claims20
A promoter comprising:
- the sequence SEQ ID NO:
- 2;
- or
- a sequence having identity greater than or equal to 90% with SEQ ID NO:
- 2.
An expression system comprising a promoter comprising:
- a sequence of SEQ ID NO:
- 2 or a sequence having identity greater than or equal to 90% with SEQ ID NO:
- 2;
- and
- a transgene placed under the control of said promoter.
A pharmaceutical composition comprising an expression system comprising a promoter comprising:
- a sequence of SEQ ID NO:
- 2 or a sequence having identity greater than or equal to 90% with SEQ ID NO:
- 2;
- and
- a transgene placed under the control of said promoter.
(canceled)
(canceled)
A method of treating a neuromuscular disease, comprising:
- administering to a subject in need a pharmaceutical composition comprising an expression system comprising a promoter comprising:
- a sequence of SEQ ID NO:
- 2 or a sequence having identity greater than or equal to 90% with SEQ ID NO:
- 2;
- and
- a transgene placed under the control of said promoter.
Dependencies are inferred from the claim text.