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Stem Cell Related Patent Number US5911983
| Title: | Gene therapy for Gaucher disease using retroviral vectors | | Inventors: | Barranger, John A.; Gibsonia, PA, USA
Robbins, Paul; Pittsburgh, PA, USA
Bahnson, Alfred B.; Pittsburgh, PA, USA | | Summary: | Described herein are therapeutic genetic methods for the treatment of Gaucher disease with retroviral vectors. The invention relates to the use of retroviral vectors which express a DNA molecule encoding the glucocerebrosidase gene, and which are applied to a process by which autologous human hematopoietic progenitor cells, namely, human CD34+ cells, are transduced with these retroviral vectors. Specific therapeutic methods are provided for transplanting the transduced cells into a Gaucher disease patient in order to provide therapeutically effective levels of glucocerebrosidase activity. | | 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. | | US Patent Website: | Click Here for Full Text of Patent | | Title Number: | US5911983 | | Application Number: | US1995000466597 | | Date Filed: | 06/06/1995 | | Date Published: | 15/06/1999 | | Assignee: | University of Pittsburgh, Pittsburgh, PA, USA |
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