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What's new in HSCI research

New gene-transport system gets more drugs to sick muscles

Harvard Stem Cell Institute (HSCI) affiliated faculty engineered a new gene delivery system that has the potential to make gene therapy for muscle diseases both safer and more effective for patients.
  • What they did: Researchers associated with the Harvard Stem Cell Institute developed a new type of adenovirus to transport therapeutic genes or the CRISPR-Cas 9 gene editing system to muscle cells to treat genetic muscle disease such as Duchenne muscular dystrophy.
  • What they found: Compared to existing approaches, this new family of adeno-associated viruses, MyoAAV, is up to 10 times more efficient at transporting these gene therapeutics to muscle cells and at much lower doses, resulting in many fewer side effects such as liver toxicity.
  • Why it matters: MyoAAV has the potential to make gene therapy for muscle diseases both safer and more effective for more patients.

A new therapy for treating Type 1 diabetes

Promising early results show that longstanding Harvard Stem Cell Institute (HSCI) research may have paved the way for a breakthrough treatment of Type 1 diabetes.
  • What they did: Using stem cells, developed a fully differentiated pancreatic islet cell replacement containing cells that accurately produce and secrete hormone insulin.
  • What they found: In a clinical trial, a patient receiving a single infusion showed robust restored function insulin secretion and blood glucose control.
  • Why it matters: This stem-cell-derived infusion could become a life-changing therapy for patients with Type 1 diabetes.
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