X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder characterized by high susceptibility to infections that results from genetic mutations in the cytochrome b-245 beta chain ...

A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient transduction of airway epithelial cells to achieve therapeutic correction. Gene editing is a method to permanently ...

CHANGE-seq-BE was developed to enable scientists to better understand base editors, an important class of CRISPR precise genome editors.

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St. Jude Children's Research Hospital. Significant amounts of time and resources ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation. The treatment was ...

A schematic showing the cytosine base editing intermediate and the various outcomes that can occur when the UNG protein is active. You may have seen it in the news recently: a baby in Pennsylvania ...

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Dr. David Liu, pictured with former lab members Holly Rees ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...