TRANSDUCTION OF GENES INTO HUMAN HEMATOPOIETIC STEM CELLS USING RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS

摘要

The invention features a method of treating a disease of hematopoietic cells in a mammal involving introducing into the mammal hematopoietic stem cells containing an adeno-associated viral vector containing a nucleic acid encoding a gene product capable of alleviating symptoms of the disease.