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Single-cell screening of cerebral organoids to identify developmental defects in autism

Researchers develop CRISPR-human organoids-single-cell RNA sequencing system to identify developmental brain defects in autism.

Dual CRISPR-Cas3 is a promising tool to induce a gigantic genomic deletion and restore dystrophin protein

Researchers evaluated the use of a dual clustered regularly interspaced short palindromic repeats (CRISPR)-Cas3 system.

Can genome editing transform ocular disease treatment?

Unveiling the key defender: PLSCR1 identified as a potent anti-SARS-CoV-2 factor in COVID-19 immunity

Genome editing prevents hypertrophic cardiomyopathy in mice

In a recent study published in the journal PLoS Digital Health, researchers pursued one-time cures for hypertrophic cardiomyopathy (HCM). They used a previously constructed murine model of HCM, designated as R403Q-129SvEv, to evaluate two different genetic therapies, as follows:

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