Efficient systemic treatment of dystrophic muscle pathologies
A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; the composition is systemically administered.
1. A method of treating Duchenne muscular dystrophy (DMD) in a human, comprising:
systemically administering to a human in need thereof a gene therapy product that comprises an adeno-associated viral (AAV) vector which harbors a nucleic acid sequence encoding a Δ4-R23/ΔCT microdystrophin, wherein the nucleic acid sequence comprises SEQ ID NO: 1, thereby treating DMD in the human.
2. The method according to claim 1 , wherein the composition is administered by intravascular injection.
3. The method according to claim 1 , wherein the AAV vector is an AAV of serotype 2, 8 or 9.
4. The method according to claim 3 , wherein the AAV vector is an AAV8 vector.
5. The method according to claim 1 , wherein the composition is administered with a dose less or equal to 10 15 vg/kg.
6. The method according to claim 1 , consisting of systemically administering the composition once.
7. The method according to claim 1 , wherein the composition is administered by intravenous injection.
8. The method according to claim 3 , wherein the AAV vector is an AAV2/8 vector.
9. The method according to claim 5 , wherein the composition is administered with a dose between 10 12 vg/kg and 10 14 vg/kg.