CMN Weekly (8 December 2023) - Your Weekly CRISPR Medicine News

Some of the best links we picked up around the internet

By: Gorm Palmgren - Dec. 8, 2023
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#CRISPRMED24

Top picks

  • Researchers at Precision BioSciences and elsewhere have presented the development of mitoARCUS, a mitochondrial-targeted nuclease that offers a promising approach for editing heteroplasmic mitochondrial DNA (mtDNA). Based on the small, highly specific ARCUS nucleases derived from I-CreI, this technology is designed to target and eliminate mutant mtDNA, specifically the common m.3243A>G mutation. It successfully removes mutant mtDNA without affecting wild-type DNA, improving mitochondrial function. As demonstrated in a xenograft mouse model using adeno-associated virus delivery, mitoARCUS shows potential as an in vivo gene-editing therapy for diseases associated with this mutation.
  • American researchers have successfully used adenosine base editors, optimised through testing over 100 guide RNAs and base editors, to target the SMN2 exon-7 mutation in spinal muscular atrophy (SMA). This approach, leveraging high-fidelity Cas9 variants, reverted the exon-7 mutation in up to 99% of patient-derived fibroblasts, significantly increasing SMN2 exon-7 transcript and SMN protein levels. This CRISPR-Cas-based method offers the potential for durable SMA treatments.

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  • While we await the FDA's first approval of a CRISPR therapy - Casgevy for sickle cell disease - a perspective in Nature looks at the next generation of CRISPR techniques. The piece emphasises that while basic CRISPR-Cas9 therapies can do some remarkable things, they represent the first generation of genome editing. Next-generation systems like base editing, prime editing and epigenome editing edit DNA with more precision and versatility and will lead to more effective and safe therapies.

News from CRISPR Medicine News

  • On Monday, we posted an explainer about prime editing. Prime editing is considered to have a desirable safety profile compared to conventional CRISPR-Cas9-mediated gene editing approaches because it allows for targeted DNA editing without intentionally generating DNA double-strand breaks.

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News: CMN Weekly (8 December 2023) - Your Weekly CRISPR Medicine News
News: CMN Weekly (8 December 2023) - Your Weekly CRISPR Medicine News
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