2013年10月24日 · Here we explain in detail how to use a human codon–optimized, nuclear localization sequence-flanked wild-type (WT) Cas9 nuclease or mutant Cas9 nickase to facilitate eukaryotic gene editing.

Cas9 (CRISPR associated protein 9, formerly called Cas5, Csn1, or Csx12) is a 160 kilodalton protein which plays a vital role in the immunological defense of certain bacteria against DNA viruses and plasmids, and is heavily utilized in genetic engineering applications. Its main function is to cut DNA and thereby alter a cell's genome.

18 小时之前 · CRISPR/Cas9-Mediated SHP-1-Knockout T cells Combined with Simvastatin Enhances Anti-Tumor Activity in Humanized-PDX HCC Model. ... (TEM) proportions and enhanced IFN-γ/Granzyme B/Perforin secretion, improving cytotoxicity against HCC lines. In humanized PDX models, SHP-1-KO T cells demonstrated superior tumor-killing activity. Transcriptomics ...

2023年8月30日 · CRISPR-Cas9 has been explored as a therapeutic agent for down-regulating target genes; the controlled delivery of Cas9 ribonucleoprotein (RNP) is essential for therapeutic efficacy and remains a challenge. Here, we report cascade dynamic assembly/disassembly of DNA nanoframework (NF) that enables the controlled delivery of Cas9 RNP.

2024年12月10日 · Pulman and colleagues demonstrate that transient delivery of Cas9 or adenine-base editor ribonucleoprotein complexes can enter photoreceptors in vivo without carrier compounds, achieving around 10% editing efficiency, a level comparable to the highest tested dose of AAV. Editing rates vary based on guide RNA efficacy and gene expression levels.

2022年9月9日 · There are reports on temperature dependent inhibition in vitro by anti-CRISPR proteins that prevent the binding of Cas9 to its target as well as temperature and light switchable Cas9 variants...

CRISPR/Cas9-mediated genome editing can be used to remove genes that encode inhibitory T-cell surface receptors, such as cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) and programmed cell death protein 1 (PD-1), to increase the effectiveness of T-cell-based immunotherapy in treating cancer.

We describe a targeted, continual multigene editing strategy that was applied to the Escherichia coli genome by using the Streptococcus pyogenes type II CRISPR-Cas9 system to realize a variety of precise genome modifications, including gene deletion and insertion, with a highest efficiency of 100%, which was able to achieve simultaneous ...

2025年2月5日 · In an attempt to develop a nanoscale CRSIPR/Cas9 delivery platform, we discovered that several biocompatible polymers, including polymalic acid (PMLA), polyglutamic acid (PGA), and polyaspartic acid (PLD), when conjugated with a trileucine (LLL) moiety, can effectively inhibit Cas9 nuclease function.

CRISPR-Cas9基因编辑技术就是通过人工设计的 sgRNA（guide RNA）来识别目的基因组序列，并引导 Cas9 蛋白酶进行有效切割 DNA 双链，形成双链断裂，损伤后修复会造成基因敲除或敲入等，最终达到对基因组DNA 进行修饰的目的。