AAV VECTOR COMPOSITIONS AND METHODS FOR GENE TRANSFER TO CELLS, ORGANS AND TISSUES

摘要

FIELD: chemistry.;SUBSTANCE: inventions relate to a method for delivering or transferring a heterologous polynucleotide sequence to a liver in a mammal and a method of treating a mammal with insufficient expression of a clotting factor or function. Present methods are performed by introducing an adeno-associated virus (AAV) vector comprising a heterologous polynucleotide sequence encoding a clotting factor and a VP1 Rh74 sequence as set forth in SEQ ID NO:1.;EFFECT: solutions allow the treatment of patients for whom the transfer of the AAV gene is unsuitable due to the existing humoral immunity to AAV, by obtaining therapeutic expression levels of the transgene in the liver at a low dose of the vector.;27 cl, 4 dwg, 2 tbl, 6 ex