3篇
您的检索式：作者名="Chengzu Long"
|
|
|
题名
|
作者
|
年代
|
出处
|
被引量
|
| 1 | Generation of a Hutchinson-Gilford progeria syndrome monkey model by base editing显示文摘Many human genetic diseases,including Hutchinson-Gilford progeria syndrome(HGPS),are caused by single point mutations.HGPS is a rare disorder that causes premature aging and is usually caused by a de novo point mutation in the LMNA gene.Base editors(BEs)composed of a cytidine deaminase fused to CRISPR/Cas9 nickase are highly efficient at inducing C to T base conversions in a programmable manner and can be used to generate animal disease models with single amino-acid substitutions.Here,we generated the first HGPS monkey model by delivering a BE mRNA and guide RNA(gRNA)targeting the LMNA gene via microinjection into monkey zygotes.Five out of six newborn monkeys carried the mutation specifically at the target site.HGPS monkeys expressed the toxic form of lamin A,progerin,and recapitulated the typical HGPS phenotypes including growth retardation,bone alterations,and vascular abnormalities.Thus,this monkey model genetically and clinically mimics HGPS in humans,demonstrating that the BE system can efficiently and accurately generate patient-specific disease models in non-human primates. | Fang Wang Weiqi Zhang Qiaoyan Yang Yu Kang Yanling Fan Jingkuan Wei Zunpeng Liu Shaoxing Dai Hao Li Zifan Li Lizhu Xu Chu Chu Jing Qu Chenyang Si Weizhi Ji Guang-Hui Liu Chengzu Long Yuyu Niu | 2020 | Protein & Cell2020，11，11： | 11 |
| 2 | God does not play dice, and neither does CRISPR/Cas9显示文摘The current dogma is that Cas9-generated double-strand breaks repaired in a template-free manner lead to imprecise insertion/deletion(indel)mutations;and that only template-mediated homology-directed repair(HDR)can facilitate precise modification.Hence,the repair products of non-homologous end joining(NHEJ)are considered heterogeneous in their outcome,and generally dismissed as random by nature.With this understanding of NHEJ it has long been used only for imprecise gene disruption;rather than deployed as a precise and predictable means for genome editing. | Chengzu Long | 2019 | National Science Review2019，6，3： | 2 |
| 3 | Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases显示文摘There are an estimated 10000 monogenic diseases affecting tens of millions of individuals worldwide.The application of CRISPR/Cas genome editing tools to treat monogenic diseases is an emerging strategy with the potential to generate personalized treatment approaches for these patients.CRISPR/Cas-based systems are programmable and sequence-specific genome editing tools with the capacity to generate base pair resolution manipulations to DNA or RNA.The complexity of genomic insults resulting in heritable disease requires patientspecific genome editing strategies with consideration of DNA repair pathways,and CRISPR/Cas systems of different types,species,and those with additional enzymatic capacity and/or delivery methods.In this review we aim to discuss broad and multifaceted therapeutic applications of CRISPR/Cas gene editing systems including in harnessing of homology directed repair,non-homologous end joining,microhomology-mediated end joining,and base editing to permanently correct diverse monogenic diseases. | Colin T.Konishi Chengzu Long | 2021 | The Journal of Biomedical Research2021，35，2： | 0 |
您所在的机构暂未开通此部分的功能，请联系管理员