Diseases, Conditions, Syndromes

Silencing of an ALS gene safely delivered to patients in new study

UMass Medical School and Massachusetts General Hospital are the first to safely treat two research participants with a synthetic microRNA, delivered into the spinal fluid, designed to silence a human disease-causing gene. ...

Diseases, Conditions, Syndromes

US scientists developing nasal spray to prevent COVID-19

Scientists at the University of Pennsylvania and the biotech firm Regeneron are investigating whether technology developed for gene therapy can be used to make a nasal spray that will prevent infection with the new coronavirus.

Genetics

Gene therapy restores hearing in deaf mice... down to a whisper

In the summer of 2015, a team at Boston Children's Hospital and Harvard Medical School reported restoring rudimentary hearing in genetically deaf mice using gene therapy. Now the Boston Children's research team reports restoring ...

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