We performed both homology-based and GUIDE-seq nomination of candidate off-target sites, which resulted in the testing of 5002 sites from four healthy donors (not shown). We then nominated additional ...

There are an estimated 6,000 Mendelian diseases, most of which still lack cures. Gene editing is an essential technology for studies aimed at generating data about disease-causing gene mutations and ...

The CRISPR entry notes both its original function, as an immune defense in bacteria and archaea, and its recent adoption as a gene editing tool. The latter part of the definition raised eyebrows for ...

The Food and Drug Administration on Friday approved two gene therapies to treat sickle cell disease, one of the which is the first CRISPR/Cas9-based treatment to win regulatory approval in the US. The ...

A new way to eradicate HIV from the body could one day be turned into a cure for infection by this virus, although it hasn’t yet been shown to work in people. The strategy uses a relatively recent ...

The world's first patient to be treated with personalized gene editing therapy is finally headed home after over 300 days in the hospital. Nine-month-old KJ Muldoon recently underwent CRISPR-based ...

A Cambridge biotech company using CRISPR gene editing to develop therapies for blood cancers said that its experimental treatment for acute myeloid leukemia proved safe in the first recipient, who is ...

Thanks to the game-changing nature of CRISPR/Cas9, the gene-editing revolution has begun in earnest. Still, Wall Street clearly has its doubts about one of the field's founding members. This extreme ...

A powerful form of DNA-editing machinery discovered in bacteria might allow us to make much bigger changes to genomes than is currently possible with CRISPR-based techniques. However, it isn’t yet ...