clinical trials, CRISPR

A first-of-its-kind gene therapy dramatically reduced misfolded protein levels in some clinical trial participants for up to six months and reduced levels in all participants for up to a year.

New technologies spur innovation in CAR T cell therapies.

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

A trial is recruiting patients to test the gene-editing technology’s ability to treat an inherited form of blindness caused by a mutation in the CEP290 gene.

The Germany-based study will test an ex vivo genome-editing therapy for the inherited blood disorder β-thalassemia.

Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

US researchers could become the first outside China to use the gene-editing technique in the clinic. 

The results of a CRISPR-Cas9 study suggest that MELK—a protein thought to play a critical role in cancer—is not necessary for cancer cell survival.

Researchers in China will use the CRISPR-Cas9 system to edit T cells extracted from patients with cancer before those cells are returned to the body to target malignant ones.