Base editing—the genome editing technique that alters base pairs using a nuclease-deficient Cas9 fused to a deaminase—was found to restart fetal hemoglobin expression in sickle cell disease (SCD) ...
A new study describes a base editing method for treating spinal muscular atrophy (SMA). This supports early signs that “single-shot” treatments from clinical trials using genome editing technologies ...
Researchers have developed a gene editing strategy that could repair the genetic problems that lead to Huntington's disease and Friedreich's ataxia. These disorders arise because of abnormal repeats ...
A team of researchers at the Broad Institute, led by gene-editing pioneer David Liu, has developed a new genome-editing strategy that could potentially lead to a one-time treatment for multiple ...
CAMBRIDGE, Mass. & MENLO PARK, Calif.--(BUSINESS WIRE)--Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based ...
Scientists at Temple University say they have gotten closer to a cure for HIV by combining a new gene editing therapy with antiretroviral drugs. The researchers say the human immunodeficiency virus ...
Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...
Genetic alterations that give rise to a rare, fatal disorder known as MOGS-CDG paradoxically also protect cells against infection by viruses. Now, scientists at the Lewis Katz School of Medicine at ...
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