Methods for genetic modification of hematopoietic progenitor cells and uses of the modified cells
First Claim
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1. 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×
- 106 transduced CD34+ hematopoietic progenitor cells per kg body weight of the subject in a total CD34+ cell population of at least 1.63×
106 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).
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Abstract
Described are compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor (HP) cells, to transduced cells and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects. The invention particularly relates to ex vivo gene therapy of HP cells for treatment or prevention of HIV infection.
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Citations
18 Claims
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1. 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×
- 106 transduced CD34+ hematopoietic progenitor cells per kg body weight of the subject in a total CD34+ cell population of at least 1.63×
106 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). - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13)
- 106 transduced CD34+ hematopoietic progenitor cells per kg body weight of the subject in a total CD34+ cell population of at least 1.63×
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14. 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 0.5×
- 106 transduced CD34+ hematopoietic progenitor cells per kg body weight of the subject in a total CD34+ cell population of at least 1.7×
106 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). - View Dependent Claims (15, 16, 17, 18)
- 106 transduced CD34+ hematopoietic progenitor cells per kg body weight of the subject in a total CD34+ cell population of at least 1.7×
Specification