Effect of CD22-directed CAR-T cells secreting anti-CD19 T cell engagers on control of leukemia progression compared to tandem anti-CD19/CD22 CAR-T cells. This is an ASCO Meeting Abstract from the 2024 ...

As genome editing therapies move through clinical trials to regulatory approval, scientists continue the quest for the holy ...

The practice of genetic modification is as old as humanity. For thousands of years, humans have bred crops, livestock and even pets that possess desirable traits. This selective process, which alters ...

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

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 ...

CRISPR functions as a programmable genetic memory system derived from bacterial immune defenses against viral infections. Guide RNA allows for rapid multiplexed targeting compared to older ...

CRISPR Cas9 genome editing has transformed the way scientists approach gene therapy, acting like precise DNA scissors that can target and repair hereditary diseases at the genetic level. This ...

We performed both homology-based and GUIDE-seq nomination of candidate off-target sites, which resulted in the testing of 5002 sites from four healthy donors (not shown). We then nominated additional ...

Northwestern scientists have developed a new nanostructure that supercharges CRISPR’s ability to safely and efficiently enter cells, potentially unlocking its full power to treat genetic diseases. By ...

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...