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 ...
This session presents two linked talks on CRISPR–Cas9 model generation and sequence validation. Christopher Raymond will discuss how direct zygote editing in mice and rats reduces timelines and costs ...
As more patients with sickle cell disease or β-thalassemia are treated with the gene-edited cellular drug exa-cel, there are more opportunities to test for potential off-target editing. The authors ...
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a gene-editing technology that allows scientists to modify DNA with unprecedented precision. Discovered in the early 2010s, CRISPR ...
Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct gene-editing experiments. The device, CRISPR-GPT, is an artificial intelligence lab ...
The CRISPR gene editing system holds tremendous promise. It has already revolutionized biomedical research by making gene editing a straightforward process. It involves using a guide RNA molecule that ...
Development of bespoke therapies has been a driving goal in medical research. This year, the announcement of the first patient treated with personalized CRISPR therapy, Baby KJ, highlights this ...
Gene-editing techniques such as CRISPR-Cas9 have many uses in the area of food and agriculture. They can combat persistent drought and disease, and improve the colour and nutritional content of food ...
Immunotherapeutic strategies have made huge strides in cancer treatment for many solid tumors over the past decade. But the promising results have failed to make a large change in the treatment of ...
CRISPR/Cas9 technology has revolutionized genetic and biomedical research in recent years. It enables editing and modulation of gene function with an unparalleled precision and effectiveness. Among ...
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