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Precise CAG repeat contraction in a Huntington’s Disease mouse model is enabled by gene editing with SpCas9-NG

Summary by Nature
The clustered regularly interspaced palindromic repeats (CRISPR)/Cas9 system is a research hotspot in gene therapy. However, the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations. To address this issue, we recently reported an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs. Here, as a feasibility study,…

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NatureNature

Precise CAG repeat contraction in a Huntington’s Disease mouse model is enabled by gene editing with SpCas9-NG

The clustered regularly interspaced palindromic repeats (CRISPR)/Cas9 system is a research hotspot in gene therapy. However, the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutations. To address this issue, we recently reported an engineered SpCas9 nuclease variant (SpCas9-NG) recognizing NGN PAMs. Here, as a feasibility study,…

·United Kingdom
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STATSTAT

Beam Therapeutics says CRISPR treatment shows encouraging results in lung condition

The company used base editing to fix a mutation that drives a debilitating lung condition, alpha-1 antitrypsin deficiency, or AATD.

·Boston, United States
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Endpoints NewsEndpoints News

Beam shares promising first clinical data on gene editing treatment for a lung and liver disease

Beam Therapeutics said its experimental gene editing treatment helped lower levels of mutant proteins and increase levels of corrected proteins in nine people with a lung disease called alpha-1 antitrypsin deficiency ...

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STAT broke the news in Boston, United States on Monday, March 10, 2025.
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