Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (CPVT)
The present invention concerns a method for the treatment of recessive Catecholaminergic Polymorphic Ventricular Tachycardia comprising delivering a gene into a cardiac cell.
1. A method for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia comprising: delivering a CASQ2 gene into a cardiac cell, wherein the CASQ2 gene encodes a calsequestrin protein having a sequence with 95% or more homology with the sequence of a protein encoded by the nucleotide sequence as set forth in SEQ ID NO:1 or SEQ ID NO:2, and wherein said delivery of a CASQ2 gene restores the intracellular Ca 2+ storage and Ca 2+ fluxes and has the effect of alleviating recessive catecholaminergic polymorphic ventricular tachycardia.
2. The method according to claim 1 , wherein the CASQ2 gene is inserted in a vector.
3. The method according to claim 1 , wherein the CASQ2 gene is inserted in a viral vector.
4. The method according to claim 1 , wherein the CASQ2 gene is inserted in a serotype 9 adeno-associated viral vector.
5. The method according to claim 1 , wherein the CASQ2 gene encodes a calsequestrin protein having a sequence with 95% or more homology with the sequence of a protein encoded by the nucleotide sequence as set forth in SEQ ID NO: 1 .
6. The method according to claim 1 , wherein the CASQ2 gene encodes a calsequestrin protein having a sequence with 95% or more homology with the sequence of a protein encoded by the nucleotide sequence as set forth in SEQ ID NO:2.
7. The method according to claim 1 , wherein the CASQ2 gene encodes a calsequestrin protein having a sequence encoded by the nucleotide sequence as set forth in SEQ ID NO:1.
8. The method according to claim 1 , wherein the CASQ2 gene encodes a calsequestrin protein having a sequence encoded by the nucleotide sequence as set forth in SEQ ID NO:2.
9. The method according to claim 1 , wherein the CASQ2 gene comprises a sequence having 95% or more homology with the nucleotide sequence as set forth in SEQ ID NO:1.
10. The method according to claim 1 , wherein the CASQ2 gene comprises a sequence having 95% or more homology with the nucleotide sequence as set forth in SEQ ID NO:1.