CRISPR safety check evaluates intended and unintended mutations

A team of researchers led by Professor Akitsu Hotta (Department of Clinical Application) developed a comprehensive framework ...
The New England Journal of Medicine

Specificity of CRISPR-Cas9 Editing in Exagamglogene Autotemcel

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 ...
Nature

Cas9 senses CRISPR RNA abundance to regulate CRISPR spacer acquisition

A cornerstone of CRISPR–Cas prokaryotic adaptive defence is adaptation, in which snippets of invader DNA are incorporated into the host CRISPR loci as new immune memories called spacers 1. Following ...
The New England Journal of Medicine

CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy

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 ...
Nature

CRISPR/Cas9 therapeutics: progress and prospects

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently ...

Biotech visionary is skeptical about AI’s impact on medical innovation

The inventor of the groundbreaking Crispr gene-editing technology is skeptical about artificial intelligence replacing human ...
techtimes

CRISPR Cas9 Genome Editing Explained: Designer Babies Ethical Dilemmas & DNA Ethics

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 ...
GEN

CRISPR-Cas9 Treatment of Rare Vascular Disease, MSMDS

Development of bespoke therapies has been a driving goal in medical research. This year, the announcement of the first patient treated with personalized CRISPR therapy, Baby KJ, highlights this ...
Los Angeles Times

How CRISPR Works: Unpacking the Science of Cas9 and Gene Modification

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 ...
Science Daily

Gene editing via CRISPR/Cas9 can lead to cell toxicity and genome instability, study finds

Researchers have identified critical spots on the genome where gene editing could cause an unwanted response, and they provide recommendations for safer approaches. CRISPR/Cas9 is a commonly used, ...
The Scientist

CRISPR Therapy: Progress and Prospects

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 ...

Sickle cell breakthrough: World's first CRISPR therapy cures Louisiana man's lifelong disease

A 23-year-old man from Louisiana has become one of the first people in the United States to be functionally cured of sickle ...