CRISPR/Cas9基因编辑技术因细胞内递送困难受限，本研究通过调控离子强度使Fe3O4纳米簇兼具精准尺寸和磁响应特性，构建磁纳米 ...

本研究针对镰状细胞病（SCD）基因治疗中不同策略的优劣尚不明确的问题，研究人员在免疫缺陷小鼠模型中系统比较了CRISPR-Cas9编辑、慢病毒转导和碱基编辑三种方法的疗效。结果表明，碱基编辑和慢病毒转导在降低红细胞镰变方面显著优于CRISPR-Cas9，为SCD的临床 ...

We performed both homology-based and GUIDE-seq nomination of candidate off-target sites, which resulted in the testing of 5002 sites from four healthy donors (not shown). We then nominated additional ...

Crispr Therapeutics, a leader in CRISPR/Cas9 biotechnology, recently received FDA and EMA approval for Casgevy, the first CRISPR/Cas9-based treatment for TDT and SCD. The partnership with Vertex ...

Stay up to date on the latest science with Brush Up Summaries.

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...

Scientists introduced CRISPR to the world as a gene-editing tool in summer 2012, when landmark papers from two independent groups demonstrated how the system could be wielded to make cuts in DNA. Now, ...

WEST LAFAYETTE, Ind. — More than a decade ago, scientists harnessed a bacterial molecular machine that identifies and cuts specific sections of DNA, revolutionizing the ability to edit genes and ...

Researchers have now used a gene-drive system to suppress an important agricultural pest. Researchers have developed a "homing gene drive system" based on CRISPR/Cas9 that could be used to suppress ...

Non-alcoholic fatty liver disease (NAFLD) is characterized by an excessive accumulation of lipids in hepatocytes in non-alcoholic individuals. NAFLD progressively changes from fatty liver to ...