Melissa Spencer, PhD, discussed the rare and complex landscape of limb girdle muscular dystrophy, highlighting research challenges, safety considerations in gene therapy, and the need for natural history studies and biomarkers.
Melissa Spencer, PhD
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Articles by Melissa Spencer, PhD
The professor in residence at UCLA Health discussed the critical considerations when deciding appropriate patients for gene therapies, specifically AAV vectors, in treating muscular dystrophies. [WATCH TIME: 4 minutes]
The professor in residence at UCLA Health provided clinical insight on the limitations of certain gene therapy vehicles for muscular dystrophies, and the need for better vectors that are more myotropic. [WATCH TIME: 3 minutes]
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