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 ...
Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein. The combination of gene editing and patient-specific stem cells may offer a new ray of ...
Researchers at the Broad Institute of MIT and Harvard have developed a gene-editing treatment for prion disease that extends ...
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 ...
Spinal muscular atrophy is a leading genetic cause of infant death worldwide, and is typically caused by loss-of-function mutations in the SMN1 gene, which produces the SMN protein Gene therapy and ...
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 ...
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