A compact Cas12f nuclease shows high editing efficiency in human cells, with structural insights enabling an engineered variant potentially suited for future AAV‑compatible delivery.

The immune system's B cells create antibodies that can mount a response against just about anything—either destroying a pathogen or instructing the rest of the immune system to go after the offender.

For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...

Add Yahoo as a preferred source to see more of our stories on Google. (WJW) – Breakthrough treatments for sickle cell disease have been approved by the U.S. Food and Drug Administration (FDA). On Dec.

Cross-species research shows that RNA splicing patterns, not just gene activity, track maximum lifespan in mammals, revealing ...

Sickle cell disease (SCD) is a group of blood disorders, including sickle cell anaemia, that is caused by stem blood cells producing mutated red blood cells. This leads to health complications, as the ...

Now, following up on that success, a large Chinese collaboration has followed up with a description of an improved gene ...

A breakthrough treatment for sickle cell patients could soon become the first gene-editing treatment to be approved by the Food and Drug Administration (FDA). Sickle cell disease (SCD) is an inherited ...

A team of interdisciplinary researchers has developed techniques to improve the efficiency of CRISPR-Cas9, the genome editing technique that earned the Nobel Prize in 2020. A Penn State-led team of ...

New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a genetic blood disorder with few curative options. Conducted as part of the ...

At age 45, Dr. Lakiea Bailey said, for the longest time, that she was the oldest person with sickle cell anemia that she knew. The executive director of the nonprofit patient advocacy group the Sickle ...