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 ...
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) ...
Researchers develop a single genome-editing strategy to treat multiple disorders caused by nonsense mutations, promising efficient and cost-effective therapies.
Want to keep the riffraff out of the gene pool party? Sneak in and slam the gate before they arrive. That’s the central idea of a new strategy by Rice University scientists who seek to avoid ...
Professor Saar Gill and Professor Carl June from the Perelman School of Medicine at the University of Pennsylvania published a research paper titled: Epitope base editing CD45 in hematopoietic cells ...
Want to keep the riffraff out of the gene pool party? Sneak in and slam the gate before they arrive. That’s the central idea of a new strategy by Rice University scientists who seek to avoid ...
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