Advances in Stem Cell Therapy for Huntington’s Disease

Huntington's protein

Scientists at the University of California Davis’ Institute for Regenerative Cures are utilizing mesenchymal stem cells [the same type of stem cells found in teeth] to develop a new therapy that targets the genetic abnormality in Huntington’s disease. The principal investigator of the study, and the director of UC Davis stem cell program and the UC Davis Institute for Regenerative Cures, Jan Nolta said, “Our team has made a breakthrough that gives families affected by this disease hope that genetic therapy may one day become a reality.” The treatment seeks to address the root cause of the disease as opposed to merely mitigating the symptoms of the disease.

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Rare Genetic Diseases Cured with Stem Cells

Translational Genomics schematic: therapy gene inserted into lentiviral vector, replaces defective gene in blood stem cells, stem cells infused with patient's blood where they expand

Recently, researchers utilized stem cells to successfully treat six children with rare genetic diseases. Three of the children were born with Metachromatic Leukodystrophy (MLD), a hereditary neurodegenerative disease. The other three children were born with Wiskott-Aldrich Syndrome (WAS), a hereditary immune system disorder.

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