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AssigneeJohnson & Johnson Research Pty. Limited
InventorsGeoffrey P. Symonds, Rafael G. Amado, Lun -Quan Sun, Janet L. MacPherson, Gregory C. Fanning, Wayne Gerlach
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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.
A composition comprising a pharmaceutically acceptable carrier and at least 1.63×106 CD34+ hematopoietic cells per kg of body weight of a human subject to whom the composition is to be administered, at least 0.52×106 of such CD34+ hematopoietic cells being transduced with a viral construct which expresses an anti-HIV agent.
A composition comprising a pharmaceutically acceptable carrier and at least 1.63×106 CD34+ hematopoietic cells per kg of body weight of the subject to whom the composition is to be administered, at least 0.52×106 of such CD34+ hematopoietic cells being transduced with a viral construct which expresses an anti-HIV agent,
A composition comprising a pharmaceutically acceptable carrier and at least 1.63×106 CD34+ hematopoietic cells per kg of body weight of the human subject to whom the composition is to be administered, at least 0.52×106 CD34+ of such CD34+ hematopoietic cells being transformed with a gene of interest not found in the CD34+ cells prior to transformation.
A composition comprising a pharmaceutically acceptable carrier and at least 1.63×106 CD34+ hematopoietic cells per kg of body weight of a human subject to whom the composition is to be administered, at least 0.52×106 CD34+ of such CD34+ hematopoietic cells being transformed with a gene of interest not found in the CD34′ cells prior to transformation,
A method of inserting into hematopoietic cells of a human subject a gene of interest comprising:
The method of claim wherein in step 21, if the total number of CD34+ hematopoietic cells after step d) is less than 1.63×106 cells per kg of body weight of the human subject, then further including a step of cryogenically storing the CD34+ hematopoietic cells from step d), repeating steps a)-d), and combining any cryogenically stored cells with the cells from step d).
A method of inserting into hematopoietic cells of a human subject a gene of interest comprising:
A method of inserting into hematopoietic cells of a human subject a gene that expresses a ribozyme comprising nucleotides having the sequence 5′-UUA GGA UCC UGA UGA GUC CGU GAG GAC GAA ACU GGC UCC-3′ comprising:
A use of a composition comprising a pharmaceutically acceptable carrier and at least 1.63×106 CD34+ hematopoietic cells per kg of body weight of a human subject to whom the composition is to be administered, at least 0.52×106 CD34+ of such cells per kg being transduced with a viral construct which expresses an anti-HIV agent, for the manufacture of a medicament for the treatment of the human subject infected with HIV.
A kit comprising
Dependencies are inferred from the claim text.