Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.

Understanding human gene function in living organisms has long been hampered by fundamental differences between species.

Picture CRISPR-Cas9, a gene editing technology, as a GPS-guided scalpel: gRNA directs the Cas9 enzyme, a protein that cuts ...

CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...

In 2020, Jennifer Doudna, Ph.D., received the Nobel Prize in Chemistry, CRISPR-Cas9, a method for genome editing. Often referred to as “molecular scissors,” CRISPR cuts DNA at specific locations that ...

Illustration showing how autophagy induction increases the efficiency of precise gene editing. In normal cells, DNA breaks caused by CRISPR–Cas9 are frequently repaired through non-homologous end ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...

A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized gene-editing therapy. Results of the groundbreaking treatment have been published ...

Gene editing has made huge leaps in recent years, such as treating the congenital blood disorders sickle cell anemia and beta thalassemia, which can require lifelong blood transfusions. But scientists ...

Gene editing is rewriting the future of medicine, as seen in the case of a baby named KJ Muldoon who got a fully personalised ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study, ...