This event at Harvard Medical School is free but registration is required.

Science Shaping Our World (SHOW) Development of Gene Therapy for Single Gene Disorders

6pm: Harvard Medical School , TMEC Walter Amphitheater, 260 Longwood Ave , Boston

Science Shaping Our World (SHOW) is an ongoing seminar and networking series that highlights cutting edge translational life science research and development. Our mission is to bring together a multidisciplinary audience spanning academia and industry where they can gain exposure and insight into the life science advancements changing our world. Further, we seek to promote active inter and cross-disciplinary dialogue and networking opportunities toward fostering the creation of relationships bridging bench to bedside.

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 is still evolving but is rapidly developing into new advancements at the bench, opening doors for potential applications in the clinic.