We are engaged in research which is aimed at developing biotherapeutic molecules for use in the treatment of a class of genetic diseases known as lysosomal storage diseases. We have received approval from the National Institute of Health's Recombinant DNA Advisory Committee to initiate clinical trials to treat Gaucher disease by gene therapy. This protocol will involve delivery of an enzyme (which is deficient in this disease) by retroviral transduction of bone marrow cells. Other biomolecular strategies employing recombinant DNA technology are being studied for development of treatment modalities for human genetic disease.

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
Biotechnology Resource Grants (P41)
Project #
5P41RR006009-10
Application #
6220996
Study Section
Project Start
1999-08-01
Project End
2000-07-31
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
10
Fiscal Year
1999
Total Cost
Indirect Cost
Name
Mellon Pitts Corporation (Mpc Corp)
Department
Type
DUNS #
City
Pittsburgh
State
PA
Country
United States
Zip Code
15213
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