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) ...

Scientists at St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard found base editing increased fetal hemoglobin production in a new treatment for sickle cell disease and ...

In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the ...

Both CBEs and ABEs have been extensively engineered to improve their efficiency, specificity, and targeting range, enabling a wide variety of base pair conversions across the genome. Base editing can ...

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 ...

Alyssa, 13, was the first patient to receive a base-edited cell therapy to treat her leukemia in May 2022.Great Ormond Street Hospital for Children A 13-year-old girl was the world's first patient to ...

CRISPR-Cas9 gene editing relies on a guide RNA that binds to a desired DNA sequence and a Cas9 enzyme that cuts both strands of DNA at that site, creating a double-strand break. Scientists edit the ...

Two new studies offer more validation that prime editing and base editing have the potential to permanently fix a gene variant associated with the rare disease phenylketonuria. In two separate papers ...

The gene-editing method known as base editing might be a way to address the underlying cause of Hutchinson-Gilford progeria syndrome (HGPS or progeria), a rare genetic disease that leads to ...

Base editing is a novel gene editing approach that can precisely change individual building blocks in a DNA sequence. By installing such a point mutation in a specific gene, an international research ...

A 13-year-old girl was the world's first patient to get a cell therapy called base editing in May. The experimental treatment has put her leukemia in remission for six months and counting. Base ...