Director/Moderator: Jose Cancelas, MD, PhD
Speakers: Carolyn Lutzko, PhD; Donald Kohn, MD; David Largaespada, PhD
Intended Audience: Physicians, Scientists, Technologists, Nurses, Managers/Supervisors, CEOs/CFOs
· Describe how CRISPR/Cas9 based genome editing can be used to understand monogenic diseases.
· Determine how CRISPR/Cas9 genome editing of hematopoietic stem cells can be translated into novel cell therapy trials.
· Review how the use of the transposase sleeping beauty may be an alternative to viral vectors in gene therapy.
Event Description: This session will provide an update on applications of novel approaches in gene therapy and diagnosis based on state-of-the-art methods of genome editing. The session will include abundant explanation of the most successful methods of genome editing including the use of CRISPR/Cas9 and transposase methods and specific examples of their application to pre-clinical and clinical trials from recognized experts in their fields.