Gemma Biotherapeutics ("GEMMABio"), a clinical-stage, global, genetic medicines company, today announced that it has been awarded funding by the Advanced Research Projects Agency for Health (ARPA-H).

A change in just one letter in the code that makes up a cancer-causing gene can significantly affect how aggressive a tumor is or how well a patient with cancer responds to a particular therapy. A new ...

Point mutations in the lamin A (LMNA) gene cause several human diseases, ranging from congenital muscular dystrophy to premature aging, collectively known as laminopathies, which affect skeletal ...

Whole-brain genome editing to correct single-base mutations and reduce or reverse behavioral changes in animal models of autism spectrum disorder (ASD) has now been achieved. Fudan University and ...

Researchers at Fudan University and the Chinese Academy of Sciences, Shanghai, China, have conducted whole-brain genome editing targeted to correct a single-base mutation associated with autism ...

Adeno-associated virus vector containing an adenine base editor provides an effective strategy to treat hereditary hearing loss caused by the R75W mutation. Moreover, this gene editing technology can ...

While researchers have long appreciated the enormous potential of genome editing to treat disease, applications of these technologies in humans have historically been limited by safety concerns. Even ...

A change in just one letter in the code that makes up a cancer-causing gene can significantly affect how aggressive a tumor is or how well a patient with cancer responds to a particular therapy. A new ...

Prof. Lukas Dow, biochemistry, and his team published a study in Nature on Aug. 16, detailing a new, more specific gene editing tool that they created to study cancer mutations through preclinical ...