CRISPR-Cas9是一种革命性的基因编辑技术,它允许研究人员精确地在基因组中特定位置进行切割和修改。AAV6作为一种病毒载体,已被广泛用于将遗传材料传递到细胞中,因其高效且毒性较低而被青睐。 在基因组工程中,精…
他们描述了一种基于Cas9/AAV6的靶向基因组编辑策略,以产生CCR5和CXCR4嗜性的HIV-1抗性的自体HSCT,编辑后的人类造血干细胞和祖细胞(HSPC)在体内维持多谱系增殖能力,而编辑后的原代人类
Mesenchymal stem cellsHuman mesenchymal stromal cells (hMSCs) are a promising source for engineered cell-based therapies in which genetic engineering could enhance therapeutic efficacy and install novel cellular functions. Here, we describe an optimized Cas9-AAV6-based genome editing tool platform for ...
核酸酶AAV病毒(AAV6),saCas9 Nuclease AAV Virus (AAV6) 产品形式 生化试剂 保存建议 蓝冰/冰袋运输保存在4℃/-20℃,若干冰运输保存在-80℃ 其他 Applied Biological Materials Inc(abm)公司是加拿大一家不断寻找用于生命科学研究和药物开发新产品的公司。众多的产品线全面涵盖了许多尖端技术,主要包括RT-PCR,RNA...
Although several Cas9-based-correction approaches have been proposed, functional correction of in vivo erythropoiesis has not been investigated previously. Here, we use a humanized globin-cluster SCD mouse model to study Cas9-AAV6-mediatedcorrection in functional HSCs within the context of autologous ...
We also found Cas9/sgRNA ribonucleoprotein treatment to be well tolerated, in spite of eliciting a DNA damage signature. Overall, this data establishes a benchmark for cellular tolerance of CRISPR/Cas9-AAV6-based genome editing, ensuring that the clinical protocol is as safe and efficient as ...
We also found Cas9/sgRNA ribonucleoprotein treatment to be well tolerated, in spite of eliciting a DNA damage signature. Overall, this data establishes a benchmark for cellular tolerance of CRISPR/Cas9-AAV6-based genome editing, ensuring that the clinical protocol is as safe and efficient as ...
AAV6CRISPR/Cas9ESCRNPelectroporationgene targetinggenome editinghomology-directed repairiPSCsgRNAGenome editing of human pluripotent stem cells (hPSCs) provides powerful opportunities for in vitro disease modeling, drug discovery, and personalized stem cell-based therapeutics. Currently, only small edits can ...
Although several Cas9-based HBB -correction approaches have been proposed, functional correction of in vivo erythropoiesis has not been investigated. Here, we used a humanized globin-cluster SCD mouse model to study Cas9-AAV6-mediated HBB -correction in functional HSCs within the context of ...