Year: 2020
Language: English
Author(s): World Federation of Hemophilia
Date: June 15, 2020
Also Available In: Spanish, French
Access WebinarIn this session from the WFH 2020 Virtual Summit, Steve Pipe and Luigi Naldini explore the promise of gene therapy and the broad-based multi-stakeholder preparation that is needed to deliver on this achievement.
Steve Pipe explores that, although we currently stand on the threshold of this achievement, delivering on this promise will require broad-based multi-stakeholder preparation with a focused emphasis on education, approval of safe and effective therapies, removal of barriers to access and excellence in clinical delivery.
Luigi Naldini continues to investigate new strategies to overcome the major hurdles to safe and effective gene transfer and translate them into new therapeutic strategies for genetic disease and cancer. Naldini presents his data regarding the further development of lentiviral vectors as a systemic gene delivery vehicle to complement the reach of AAV vectors.
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