Gene therapyDNA repairDouble strand breakhomologous recombinationnon homologous end joiningRecent advances in CRISPR-Cas9 genome editing tool have made great promises to basic and biomedical research as well as gene therapy. Efforts to make the CRISPR-Cas9 system applicable in gene therapy are largely ...
导读:日前,发表在《Gene Therapy》上的一项最新研究中,研究人员探讨了靶定HBV抗原编码区的CRISPR/CRISPR相关Cas9系统,在细胞培养系统和活体动物体内的效果。结果表明,CRISPR/Cas9可在体内和体外抑制HBV复制和表达,可能是HBV感染一种新的治疗策略。 2月5日,来自...
近日,由北京大学邓宏魁研究组等多个研究团队首次利用CRISPR/Cas9基因编辑的成体HSCs治疗艾滋病和白血病患者,实现了CCR5突变的HSPCs在患有艾滋病合并急性淋巴细胞患者中同种异体移植,急性淋巴母细胞性白血病仅在移植后4周就得到了完全缓解。由于CRISPR/Cas9体外敲除效率偏低,移植后的受体反应效率还不足以达到治愈HIV-1的水...
At the forefront of medicine, Gene Therapy brings you the latest research into genetic and cell-based technologies to treat disease. It also publishes reviews and articles, which highlight the cutting edge of the field.
基因编辑策略主要包括CRISPR/Cas9核酸酶非同源末端连接(NHEJ)修复方式介导的基因敲除(gene disruption)策略,通过敲除突变基因,可应用于DN和GOF导致的显性遗传性疾病;以及CRISPR/Cas9介导的同源重组修复(HDR)、碱基编辑器和先导编辑器介导的基因修复(gene correction)策略,可直接纠正基因突变位点,因此显性、隐性遗传性疾病均...
2月5日,来自军事医学科学院放射与辐射医学院研究所、第四军医大学西京医院、日本京都大学和华中农业大学兽医学院等处的研究人员,在Nature旗下子刊《Gene Therapy》发表一项最新研究成果,题为“Harnessing the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated Cas9 system to disrupt th...
Therapeutic approaches based on this technology fill the preclinical pipeline, and rely on the use of viral vectors to deliver the Cas9 gene and guide RNA to a gene of interest. However, concerns regarding the safety and efficacy of CRISPR-Cas9 use in gene therapy remain. A pre-print ...
of gene expression. This study will present an overview of the primary epigenetic alterations seen in pulmonary cancer, as well as a summary of therapeutic implications for targeting epigenetics in the management of pulmonary cancer, with a particular emphasis on the technique known as CRISPR/Cas9....
With target specificity defined by a very short RNA-coding region, the CRISPR-Cas9 system greatly simplifies genome editing and has great promise in broad applications such as stem cell engineering, gene therapy, tissue and animal disease models, and engineering disease-resistant transgenic plants...
A complete CRISPR and TALEN gene editing toolkit To help researchers in their quest to understand how the genome influences phenotype, we’ve developed a complete set of trusted solutions for every step in the gene editing workflow. Design your CRISPR-Cas9 system or TALEN constructs for precise...