The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the ...

A new class of CRISPR-based tools efficiently corrects point mutations in cell lines, animal models and perhaps the clinic. When Xingxu Huang began thinking about correcting disease-causing mutations ...

In a global first, scientists have used advanced base editing to repair a deadly gene mutation in a newborn, marking a major leap forward in the treatment of rare ...

Nucleotides are the building blocks of the two nucleic acids, ribonucleic acid (RNA) and deoxyribonucleic acid (DNA). As such, they are also the building blocks of you and every other lifeform on ...

Genome editing holds promise for correcting pathogenic mutations. However, it is difficult to determine off-target effects of editing due to single-nucleotide polymorphism in individuals. Here we ...