Developing CRISPR Gene Therapies for Neurodegenerative Diseases

By American Neurological Association FEATURING Claire Clelland

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October 6, 2024

Developing CRISPR Gene Therapy for Neurodegenerative Diseases brought to you from the American Neurological Association’s 149th Annual Meeting featuring Claire Clelland, PhD, MD, Mphil.

Many neurologic diseases can be caused by mutations in single genes. Dominant mutations that cause disease through toxic gain of function mechanisms are ideal targets for CRISPR gene therapies. We are developing CRISPR gene therapies for neurodegenerative diseases including dementias and amyotrophic lateral sclerosis (ALS) starting with C9orf72, the leading genetic cause of frontotemporal dementia (FTD) and ALS.

To see more cutting-edge research presented at the ANA2024 meeting, please navigate to https://education.myana.org/

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