Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
Nov 18, 2021
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Adding to the growing body of literature demonstrating the feasibility of correcting lethal genetic diseases before birth, researchers at Children's Hospital of Philadelphia (CHOP) have used DNA base editing in a prenatal ...
Jul 13, 2021
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Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to try to treat a disease, although it's too soon to know if this will help.
Feb 7, 2019
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A new therapy involving stem cells may offer a better quality of life for patients with Hurler syndrome, a rare genetic disease that can cause crippling physical conditions and lead to early death.
Nov 16, 2017
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REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO's NAV AAV8 vectors resulted in sustained serum α-L-iduronidase (IDUA) expression, as well as correction of systemic features of MPS I, or Hurler syndrome, ...
Sep 30, 2014
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Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.
Jul 24, 2013
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Researchers have overcome a major challenge to treating brain diseases by engineering an experimental molecular therapy that crosses the blood-brain barrier to reverse neurological lysosomal storage disease in mice.
Feb 4, 2013
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