首页>
外国专利>
Peptide-modified hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced hepatic tropism and increased muscle transduction
Peptide-modified hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced hepatic tropism and increased muscle transduction
The present invention relates to a recombinant adeno-associated virus (AAV) capsid protein that is a peptide-modified hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins comprising at least one copy of a peptide comprising RGD. to which the recombinant peptide-modified hybrid AAV capsid protein has more reduced hepatic tropism and increased muscle transduction compared to the recombinant hybrid AAV capsid protein without the peptide. The present invention also relates to derivatized peptide-modified hybrid AAV serotype vector particles packaging a gene of interest and their use for gene therapy, in particular for treating neuromuscular genetic diseases, in particular muscular genetic diseases.
展开▼