Patent ID: 7345025

Claim:
A composition for engraftment of transduced autologous CD34 + hematopoietic progenitor cells in a human subject which comprises a pharmaceutically acceptable carrier and a minimum dose of 0.52×10 6 transduced CD34 + hematopoietic progenitor cells per kg body weight of the subject in a total CD34 + cell population of at least 1.63×10 6 CD34 + hematopoietic progenitor cells per kg body weight of the subject, the transduced cells comprising a RRz2 vector encoding a ribozyme and the number of transduced cells being determinable by performing competitive PCR on such transduced CD34 + hematopoietic cells, wherein the ribozyme is encoded by DNA nucleotides having the sequence 5′-TTA GGA TCC TGA TGA GTC CGT GAG GAC GAA ACT GGC TC-3′ (SEQ ID NO: 3).