For example, consider a CRISPR‐Cas9 gene knockout (CRISPR KO) screen in which a Cas9 nuclease is directed by a sequence‐programmable guide RNA (gRNA) to a complementary genomic target, generating ...

This work was supported by a V Foundation grant awarded to C.S.T. (T2022-018). S.M.L. was funded by a T32 Physical Genomics Training Fellowship (T32GM142604) and the Dr. John N. Nicholson Fellowship ...

This review introduces the fundamental concepts and mechanisms of gene editing, with a particular emphasis on CRISPR-Cas systems. The principles and methods used in the development and optimization of ...

The Modernization Engineering Technology Research Center of Ethnic Minority Medicine of Hubei Province, School of Pharmaceutical Sciences, South-Central Minzu University, Wuhan 430074, China ...

“How awesome is it that we’re at this point?” said Nessan Bermingham, who co-founded and led Intellia, one of the first CRISPR companies, until 2017.

State Key Laboratory of Biotherapy and Cancer Center/Collaborative Innovation Center for Biotherapy, Department of Pulmonary and Critical Care Medicine, Targeted Tracer Research and Development ...

Allison DeAngelis is the East Coast biotech and venture capital reporter at STAT, reporting where scientific ideas and money meet. She is also co-host of the weekly biotech podcast, The Readout ...

When a neuron in our body gets damaged, segments of RNA produce proteins that can help repair the injury. But in neurological disorders such as ALS and spinal muscular atrophy, or following spinal ...

This study set out to answer that question using cutting-edge biotechnology. The team used CRISPR-Cas9 to engineer several ALS-causing mutations—including FUS P525L, FUS R495X, and TARDBP M337V ...