genetic modification of pluripotent hemopoietic stem cells of primates by transduction of p - phsc with a recombinant adeno - associated virus . tile genomc of the recombinant aav comprises a dna sequence flanked by the inverted terminal repeats of aav . the dna sequence will normally comprise regulatory sequences which are functional in hemopoictic cells and , controlled by these regulatory sequences , a sequence coding for a protein or rna with a therapeutic property when introduced into hemopoietic cells . preferred examples of dna sequences are the human lysosomal glococerebrosidase gene , a globin gene from the human β - globin gene cluster , a dna sequence encoding an rna or protein with anti - viral activity , the α1 - antitrypsin gene and the human multidrug resistance gene i . the invention provides for effective gene therapy with phsc of primates , in particular humans .