Scientists have used a gene therapy tool that acts like intelligent molecular scissors to correct the key gene defect in mice with hemophilia B. Scientists have used a gene therapy tool that acts like ...
4monon MSN
CRISPR gene editing in blood stem cells linked to premature aging effects: Study offers solutions
Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using CRISPR-Cas9 in combination with AAV6 vectors can trigger inflammatory and ...
Editas Medicine, Inc. focuses on gene-editing using CRISPR technology to develop medicines for previously untreatable or under-treated diseases. The company's leading product, EDIT-301, aims to treat ...
In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have ...
Editas Medicine Inc EDIT announced initial safety and efficacy data from the first four patients with sickle cell disease (SCD) treated with EDIT-301 in the RUBY trial and from the first ...
Both therapies use patients’ modified blood stem cells given back as a one-time, single-dose infusion. Sickle cell disease is an iherited blood disorders affecting approximately 100,000 people in the ...
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