US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.
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Children's Hospital of Philadelphia (CHOP)
Elevated levels of a biomarker related to blood vessel damage have been found in all children with SARS-CoV-2 infection.
The prodrug developed by researchers caused long-term remission in all murine models of high-risk or drug-resistant cancers with fewer side-effects than a comparable drug.
A machine learning algorithm that predicts sites of DNA methylation could aid in the identification of disease-causing mechanisms, say researchers.
Researchers used computational tools to identify regions of the SARS-CoV-2 virus that could potentially provide a safe and long-term immune response.
A collaboration of academic institutions in the United States has identified a gene that is linked to alternative splicing changes that occur in several cancers.
A single leukemic cell has become resistant to CAR-T therapy after unknowingly engineered with the leukemia-targeting CAR lentivirus...