Whilst genome editing with CRISPR-Cas9 is effective, it runs the risk of off-target effects and excessive mutations, which can lead to toxicity. To reduce the likelihood of these occurring, ...
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 ...
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats ...
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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 ...
The Food and Drug Administration on Friday approved two gene therapies to treat sickle cell disease, one of the which is the first CRISPR/Cas9-based treatment to win regulatory approval in the US. The ...
As far back as 2015, scientists have been warning against using CRISPR-Cas9 technology to modify germline genomes. Indeed, a perspective published that year in Science suggested that otherwise ...
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 ...