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 ...

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 ...

Base editing has been lauded as a potential “game changer” for a broad swath of diseases, and Revvity is aiming to help usher in that change. After exclusively licensing a base editing system dubbed ...

(MEMPHIS, Tenn. – July 03, 2023) Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia. These two common life-threatening anemias afflict ...

A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient transduction of airway epithelial cells to achieve therapeutic correction. Gene editing is a method to permanently ...

Add Yahoo as a preferred source to see more of our stories on Google. Alyssa, 13, was the first patient to receive a base-edited cell therapy to treat her leukemia in May 2022.Great Ormond Street ...

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 ...

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 ...

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 ...