Gene Therapy : NIDDK

Gene Therapy

The Gene Therapy Program encompasses research aimed at developing basic and applied gene therapy for genetic metabolic diseases. Specific areas of support include:

  • Studies to improve gene delivery systems; 
  • Studies of the basic biology of AAV, adenovirus, retrovirus, and lentivirus vectors; 
  • Studies of nonviral methods of gene transfer such as liposomes or DNA conjugates;
  • Studies to target gene delivery to specific cell types;
  • Studies of gene therapy of stem cells to treat a genetic metabolic disease.

For further information, contact Dr. Catherine McKeon,  Senior Advisor for Genetic Research.

Resources for Researchers

Clinical Research


  • Molecular Therapy and Cystic Fibrosis Centers - Overview & Map

Related Study Sections

See Also

Office of Rare Diseases

Funding Opportunities

Funding for Special Communities



Related Organizations

Page last updated: January 20, 2009

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