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Joseph Samaha, Jim Dagher, Shayan Abdollah Zadegan

Huntington’s disease (HD) is a neurodegenerative disease inherited in an autosomal dominant manner. It is caused by an expansion of cytosine, adenine, guanine (CAG) repeats within the huntingtin (HTT) gene, which is located on chromosome 4. This pathological expansion of CAG repeats results in the production of a mutant huntingtin protein with an abnormally long polyglutamine […]

Lysogene-Academic Collaboration for GM1 Gangliosidosis

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Published Online: May 25th 2015

GM1 gangliosidosis is a rare inherited neurodegenerative disorder characterized by severe cognitive and motor developmental delays resulting in death of most patients at a very young age.

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