“Through CRISPR RNA optimization, we achieved around 59% editing at the TTR locus in our in vitro experiments. In the mouse ...

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

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

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

Humanized mouse models are essential for investigating human gene function; however, the complete replacement of mouse genes ...

The power of CRISPR became definitively clear when the first CRISPR-based gene therapy, Casgevy (exa-cel), won regulatory approvals for the treatment of sickle cell disease. But CRISPR’s applications ...

The study combines a deep learning model with CRISPR screens to control the expression of human genes in different ways -- such as flicking a light switch to shut them off completely or by using a ...

Researchers at Utah State University have reported a previously unrecognised CRISPR immune response in which a bacterial ...

SCD gene therapy approaches using CRISPR, gene therapy, and base editing show different stem cell outcomes in a mouse study.

Huntington's disease (HD) is a neurological disorder that causes progressive loss of movement, coordination and cognitive function. It is caused by a mutation in a single gene called huntingtin or HTT ...