In this episode I'm joined by senior author Dr. Stephanie Cherqui to talk about her recent publication in the New England Journal of Medicine on gene therapy for cystinosis. This is a very personal episode for me, because two of my children have cystinosis. The article is linked below. Thanks for listening!

Hematopoietic Stem Cell Gene Therapy for Cystinosis

Donate to the Cystinosis Research Foundation

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