Cassandra Gorsuch, PhD, on the Durability and Next Steps for the ARCUS Gene Editing Platform in DMD
April 28th 2025The chief scientific officer at Precision Biosciences discussed the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy.
Cassandra Gorsuch, PhD, on the ARCUS Gene Editing Platform and Its Potential in DMD
April 27th 2025The chief scientific officer at Precision Biosciences discussed the ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy.
OPMD and Advancements in Genetic Research
April 22nd 2025Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.
Matthew Wicklund, MD, on OPMD and Advancements in Genetic Research
April 13th 2025The professor of neurology at the University of Texas Health Science Center San Antonio discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.
Urgency of Funding and Equity in Neuromuscular Research and Care
March 31st 2025Robert Califf, MD, MACC, a cardiologist and former FDA commissioner, discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.
Robert M Califf, MD, MACC, on Urgency of Funding and Equity in Neuromuscular Research and Care
March 28th 2025The former FDA commissioner discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.
Donovan Decker’s Journey in Gene Therapy and Advocacy for LGMD—Breaking Barriers
March 24th 2025Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, discussed his powerful journey as a patient advocate and gene therapy pioneer, shedding light on challenges and progress in LGMD.
Roadblocks Faced by Gene Therapies for Muscular Dystrophies
March 14th 2025John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.
Gene and Cell Therapy Approaches Hold Promise for Usher Syndrome
September 21st 2024In honor of Usher Syndrome Awareness Day, CGTLive® interviewed Zheng-Yi Chen, DPhil, associate professor, Otolaryngology–Head and Neck Surgery, Harvard Medical School, about the current state of research in this rare disease.