Safe vector for gene therapy

Details Safe vector for gene therapy Safe vector for gene therapy

1991-11-08

1993-10-12

Schwartz, Richard A.

Chemistry: molecular biology and microbiology

Virus or bacteriophage, except for viral vector or...

4352402, 4353201, C12N 701, C12N 1586

Patent

active

052524795

ABSTRACT:
Gene therapy involves the transfer and stable insertion of new genetic information into cells. The present invention is directed to safe vectors for gene therapy and thus provides hybrid parvovirus vectors which are capable of site-specific integration into a mammalian chromosome without substantial cytotoxicity, and which direct erythroid cell-specific expression of heterologous genes. The hybrid vector is useful in gene therapy, particularly in the treatment of hemoglobinopathies and other hematopoietic diseases. A method of delivery of constitutive levels of a pharmaceutical product and a method of producing a recombinant protein are also provided.

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