Agenda Details | ASGCT Annual Meeting

Detailed Program

Thursday, May 18 2023
08:00 AM – 09:45 AM PT

Recent Advances in the Therapy of Musculo-skeletal and Neuromuscular Diseases

Muscle and bone, the bulkiest tissues in the body, present unique challenges for gene therapy. While restoring mutant genes has been a mainstay of gene therapy approaches targeting these tissues, this session will explore new delivery approaches, gene targets and cellular environment on treating muscle, bone and neurological deficits associated with musculoskeletal diseases.

Session Details

08:00 AM – 09:45 AM - Room 408 AB

Co-Chair

John T. Gray, PhD. Vertex Cell and Genetic Therapies, Boston, MA

08:00 AM – 09:45 AM - Room 408 AB

Co-Chair

Chunping Qiao, PhD. Novartis, NC

08:01 AM – 08:25 AM - Room 408 AB

Opportunities and Challenges in Pursuing Gene Therapy for Charcot-Marie-Tooth Diseases

Marina Stavrou. The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus

08:25 AM – 08:50 AM - Room 408 AB

WNT-modulating Gene Silencers as a Gene Therapy for Osteoporosis, Bone Fracture, and Critical-sized Bone Defects

Jae-hyuck Shim, PhD. UMass Chan Medical School, Worcester, MA

08:50 AM – 09:15 AM - Room 408 AB

Alternatives in the Delivery of the CRISPR-Cas9/sgRNA System to Skeletal Muscle Tissues, IPS Cells

Akitsu Hotta, PhD. Kyoto University, Japan

09:15 AM – 09:40 AM - Room 408 AB

Modeling FSHD in Mouse Therapeutic Interventions Targeting Muscle and Environment

Michael Kyba, Ph. D.. University of Minnesota, Minneapolis, MN