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SHOW March 2011

posted Mar 6, 2012, 2:11 PM by Steven Munevar   [ updated Dec 27, 2012, 11:05 AM ]

Development of Gene Therapy for Single Gene Disorders

Keynote Speaker:
Terry Flotte, M.D
  • Celia and Isaac Haidak Distinguished Professor
  • Dean, Provost and Executive Deputy Chancellor University of Massachusetts Medical School

March’s SHOW will focus on the development and application of gene therapy for the treatment of single gene disorders. The presentation will focus on efforts to direct gene therapy to target sites including recent phase 2 clinical trial results aimed at treating genetic emphysema due to alpha-1 antitrypsin deficiency.


Gene therapy based therapeutic strategies involve the insertion of functional genes into patients’ cells in order to replace a mutated or otherwise faulty gene. Alternatively, gene therapy can also involve directly correcting the mutations or modifying normal genes that may enable a viral infection or microbial pathogenicity. Gene therapy based therapeutics are still evolving but are rapidly developing into new advancements at the bench, opening doors for potential applications in the clinic.

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