Spotlighting Rare Diseases Research Blog

A new gene therapy approach for Sandhoff disease developed by UMass Chan researcher Miguel Sena-Esteves is featured in a story in USA Today.

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Two research scientists from UMass Chan Medical School — Kevin Donahue, MD and Marian Walhout, PhD — have been awarded funds for research on much-needed therapies for certain rare diseases...

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I began working in the area of rare disease research during the early years of my research training as a PhD student. This work was focused on molecular genetics, disease gene cloning, and genetic diagnosis of several rare disorders of the central nervous system (CNS), including Canavan disease, Hurler syndrome, and Hunter syndrome...

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