Now, Musunuru and colleagues are preparing a master clinical protocol to study a CRISPR therapy that's mostly the same as the one they used to treat baby KJ, except they plan to swap out the gene ...

CRISPR has taken the bioengineering world by storm since its first introduction. From treating sickle cell diseases to creating disease-resistant crops, the technology continues to boast success on ...

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 ...

Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection (tearing) and death in childhood. Currently ...

Genetic editing holds promise to treat incurable diseases, but the most popular method - CRISPR - sometimes does more harm than good. A new study from University of California San Diego and Yale ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

Base editors are a type of gene editing tool designed to make precise changes to DNA at the single-nucleotide level. They are used to correct genetic variants that cause disease. 1 This article ...

Genetic editing holds promise to treat incurable diseases, but the most popular method — CRISPR — sometimes does more harm than good. A new study from University of California San Diego and Yale ...

Out of the many challenges scientists face in bringing genomic medicine to patients, delivery is near the top of the list. Due to these barriers in getting the genetic payload to the correct target ...

Last August, KJ Muldoon was born with a potentially fatal genetic disorder. Just six months later, he received a Crispr treatment designed just for him. Muldoon has a rare disorder known as CPS1 ...