Gene therapy for Gaucher disease using retroviral vectors

Details Gene therapy for Gaucher disease using retroviral vectors Gene therapy for Gaucher disease using retroviral vectors

1995-06-06

1999-06-15

Guzo, David

Drug, bio-affecting and body treating compositions

Whole live micro-organism, cell, or virus containing

Genetically modified micro-organism, cell, or virus

424 936, 4353201, 435372, A61K 4800, C12N 510, C12N 1586

Patent

active

059119837

ABSTRACT:
The present invention relates to gene therapy for Gaucher disease using retroviral vectors which express the glucocerebrosidase gene. Methods are provided for transduction of autologous hematopoietic stem cells (e.g., human CD34+ cells) with these vectors and for transplantation of the transduced cells into a Gaucher disease patient to provide therapeutically effective levels of glucocerebrosidase activity. The invention also provides for retroviral vectors that express the glucocerebrosidase gene, and for human hematopoietic cells that contain the retroviral vector.

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