期刊文献+
任意字段
题名或关键词
题名
关键词
摘要
作者
第一作者
机构
刊名
分类号
参考文献
作者简介
基金资助
栏目信息
共找到242篇文章
< 1 2 … 13 >
每页显示 20 50 100
已选0条
引用分析
参考文献
引证文献
 统计分析
检索结果
已选文献
显示方式：
Nanoparticle-assisted gene editing for genomic disorders in the central nervous system 认领 引用
1
作者 Hyeon-Yeol Cho Jeong-Woo Choi 《Neural Regeneration Research》 SCIE CAS CSCD 2026年第8期3547-3548,共2页
Genomic disorders affecting the central nervous system(CNS)are among the most complex and devastating conditions in human health.Moreover,these disorders,such as Rett syndrome,spinal muscular atrophy,and Fragile X syn... Genomic disorders affecting the central nervous system(CNS)are among the most complex and devastating conditions in human health.Moreover,these disorders,such as Rett syndrome,spinal muscular atrophy,and Fragile X syndrome,are typically caused by mutations in genes essential for neural development,synaptic function,or cellular homeostasis.Despite the genetic diversity involved,these diseases share key pathological features,including progressive neurodegeneration,disruption of neural circuits,and loss of cognitive or motor function.Meanwhile,one of the significant clinical challenges in treating CNS disorders is the limited regenerative capacity of the adult nervous system,which makes reversing disease progression extremely difficult once symptoms appear.In addition,the blood-brain barrier(BBB)restricts the passage of most systemically administered therapeutics,further complicating effective intervention.Consequently,current treatment options remain largely palliative,and effective cures remain elusive. 展开更多
关键词 nanoparticle assisted gene editing central nervous system genomic disorders fragile x syndromeare central nervous system cns rett syndromespinal muscular atrophyand progressive neurodegenerationdisruption neural circuitsand loss cogni
暂未订购 下载PDF
Generating popcorn-like fragrant tomato using CRISPR/Cas9-mediated gene editing 认领 引用
2
作者 Peng Zheng Wei Jiang +8 位作者 Qionglin Chen Shouli Feng Miaomiao Huang Lu Yuan Lingyun Chen Xiaoyuan Tao Zhong-Hua Chen Jingyin Yu Shengchun Xu 《Journal of Integrative Agriculture》 SCIE CAS CSCD 2026年第5期2174-2177,共4页
Tomato(Solanum lycopersicum L.) is one of the world's most widely cultivated and consumed vegetables,and its fruits are highly valued for its vibrant color,culinary versatility,and nutritional benefits(Yu et al.20... Tomato(Solanum lycopersicum L.) is one of the world's most widely cultivated and consumed vegetables,and its fruits are highly valued for its vibrant color,culinary versatility,and nutritional benefits(Yu et al.2025).China leads global tomato production,with an output of approximately 70 million tons in 2023,contributing to a worldwide yield exceeding192 million tons(FAO 2025).Fragrance significantly enhances the sensory appeal and flavor quality of food,thereby influencing the consumption and market price of agricultural products.For example,fragrant rice(Oryza sativa L.) varieties like Basmati and Jasmine have gained increasing popularity worldwide due to their distinct aroma,and their prices are often more than twice that of regular rice(Shan et al.2015).Among the more than 200 volatile organic compounds(VOCs) characterized in aromatic rice,2-acetyl-1-pyrroline(2-AP) is a dominant VOC responsible for the delightful "popcorn-like" aroma(Okpala et al.2019). 展开更多
关键词 tomato fragrance volatile organic compounds CRISPR Cas Solanum lycopersicum popcorn aroma gene editing acetyl pyrroline
暂未订购 下载PDF
Wolfram syndrome:A perspective on gene editing as a therapeutic strategy 认领 引用
3
作者 Steven Bergmans Lies De Groef 《Neural Regeneration Research》 SCIE CAS CSCD 2026年第7期2954-2955,共2页
Wolfram syndrome(WS)is a rare autosomal rece s s i ve disease characte r i zed by the development of diabetes insipidus,diabetes mellitus,optic atrophy,and deafness(often referred to as DIDMOAD),and overall severe neu... Wolfram syndrome(WS)is a rare autosomal rece s s i ve disease characte r i zed by the development of diabetes insipidus,diabetes mellitus,optic atrophy,and deafness(often referred to as DIDMOAD),and overall severe neurodegenerative fallback.The global prevalence of this disease is estimated at 1 in 770,000(Lee et al.,2023).It is most commonly caused by biallelic(point)mutations in the Wolframin endoplasmic reticulum(ER)transmembrane glycoprotein(WFS1)gene(in case of WS type 1),but mutations in the CDGSH Iron Sulfur Domain 2(CISD2)are also linked to WS(type 2).The latter,however,often present with less severe pathological manifestations(Lee et al.,2023).WFS1 is located on chromosome 4p16.1 and spans over 33 kilobases.Many mutation variants have been identified in WFS1,encompassing missense,nonsense,and frameshift mutations.These mutations are spread across the coding region of WFS1,but certain regions,such as exon 8,the largest exon,appear particularly mutation-prone and associated with the classical WS type 1 phenotype(Lee et al.,2023). 展开更多
关键词 Therapeutic Strategy wolfram syndrome ws autosomal rece s s i ve disease Wolfram Syndrome Diabetes Insipidus wolframin endoplasmic reticulum er transmembrane glycoprotein wfs gene cdgsh iron Gene Editing
暂未订购 下载PDF
Harnessing Agrobacterium rhizogenes and mobile elements for innovative transgene-free gene editing in woody plants 认领 引用
4
作者 Hui Duan Bin Wu +1 位作者 Hongmin Qin Margaret R Pooler 《Ornamental Plant Research》 2025年第1期188-192,共5页
Woody landscape plants are an important sector of US agriculture and play critical roles in the urban landscape by adding value to residential,public,and commercial properties.Genetic improvement of woody landscape pl... Woody landscape plants are an important sector of US agriculture and play critical roles in the urban landscape by adding value to residential,public,and commercial properties.Genetic improvement of woody landscape plants is slower than that of most other crops because of long juvenility periods,heterozygosity,and resources required to grow out large populations.New breeding technologies,especially gene editing,hold great promise to complement and improve traditional breeding.This is particularly true in woody ornamental crops where the creation of transgene-free gene-edited plants would accelerate the breeding process and reduce the regulatory burden inherent with transgenic crops.In this prospective review,we propose a method to harness the power of two technologies to transform the process of woody plant gene editing.Agrobacterium rhizogenes can induce transgenic hairy roots in many plant species,while TLS(tRNA-like sequence)has been shown to facilitate the long-distance movement of transcripts from the root to the grafted scion and remain functional.We propose using A.rhizogenes to transform root tissue with mobile gene-editing components.These components would then migrate from the roots into the shoots and leaves of the plant,eliminating the need for grafting.Since only RNA,and not DNA,migrates into the upper portion of the plant,the resulting gene-edited shoot will stay transgene-free and can be propagated through either sexual or asexual methods. 展开更多
关键词 urban landscape gene editinghold transgene free gene editing woody plants Agrobacterium rhizogenes woody landscape plants gene editing technologies mobile elements
暂未订购 下载PDF
Tissue culture-independent approaches to revolutionizing plant transformation and gene editing 认领 引用 被引量：2
5
作者 Luis Felipe Quiroz Moman Khan +4 位作者 Nikita Gondalia Linyi Lai Peter C.McKeown Galina Brychkova Charles Spillane 《Horticulture Research》 SCIE CSCD 2025年第2期294-302,共9页
Despite the transformative power of gene editing for crop improvement,its widespread application across species and varieties is limited by the transformation bottleneck that exists for many crops.The genetic transfor... Despite the transformative power of gene editing for crop improvement,its widespread application across species and varieties is limited by the transformation bottleneck that exists for many crops.The genetic transformation of plants is hindered by a general reliance on in vitro regeneration through plant tissue culture.Tissue culture requires empirically determined conditions and aseptic techniques,and cannot easily be translated to recalcitrant species and genotypes.Both Agrobacterium-mediated and alternative transformation protocols are limited by a dependency on in vitro regeneration,which also limits their use by non-experts and hinders research into non-model species such as those of possible novel biopharmaceutical or nutraceutical use,as well as novel ornamental varieties.Hence,there is significant interest in developing tissue culture-independent plant transformation and gene editing approaches that can circumvent the bottlenecks associated with in vitro plant regeneration recalcitrance.Compared to tissue culture-based transformations,tissue culture-independent approaches offer advantages such as avoidance of somaclonal variation effects,with more streamlined and expeditious methodological processes.The ease of use,dependability,and accessibility of tissue culture-independent procedures can make them attractive to non-experts,outperforming classic tissue culture-dependent systems.This review explores the diversity of tissue culture-independent transformation approaches and compares them to traditional tissue culture-dependent transformation strategies.We highlight their simplicity and provide examples of recent successful transformations accomplished using these systems.Our review also addresses current limitations and explores future perspectives,highlighting the significance of these techniques for advancing plant research in crop improvement. 展开更多
关键词 plant transformation transformation bottleneck aseptic techniquesand plant tissue culturetissue culture vitro regeneration gene editing genetic transformation plants tissue culture independent
暂未订购 下载PDF
Precision Pharmacology in Pediatric Congenital Heart Disease:Gene Editing and Organoid Models Addressing Developmental Challenges 认领 引用
6
作者 Jun He Jianli Luo +2 位作者 Yanling Wang Dai Zhou Shuanglin Xiang 《Congenital Heart Disease》 SCIE 2025年第5期613-623,共11页
Pediatric congenital heart disease(CHD)pharmacotherapy faces three fundamental barriers:developmental pharmacokinetic complexity,anatomic-genetic heterogeneity,and evidence chain gaps.Traditional agents exhibit critic... Pediatric congenital heart disease(CHD)pharmacotherapy faces three fundamental barriers:developmental pharmacokinetic complexity,anatomic-genetic heterogeneity,and evidence chain gaps.Traditional agents exhibit critical limitations:digoxin’s narrow therapeutic index(0.5–0.9 ng/mL)is exacerbated by ABCB1 mutations(toxicity risk increases 4.1-fold),furosemide efficacy declines by 35%in neonates due to NKCC2 immaturity,andβ-blocker responses vary by CYP2D6 polymorphisms(poor metabolizers require 50–75%dose reduction).Novel strategies demonstrate transformative potential—CRISPR editing achieves 81%reversal of BMPR2-associated pulmonary vascular remodeling,metabolically matured cardiac organoids replicate adult myocardial energy metabolism for drug screening,and SGLT2 inhibitors activate triple mechanisms(calcium overload mitigation,mitophagy,fibrosis reversal).However,clinical translation requires overcoming developmental barriers:age-dependent enzyme expression(infant CYP2D6=30–60%adult activity),post-Fontan hepatotoxicity(bosentan trough concentrations elevates 1.8-fold),and AI model limitations(32%error in complex CHD).Future integration of placental transfer models,disease-specific organoids,and multi-omics mapping of FOXO/CRIM1 pathways will shift paradigms from symptom control to curative repair. 展开更多
关键词 Congenital heart disease molecular pathogenesis precision medicine gene editing translational challenges
暂未订购 下载PDF
Application and Prospects of CRISPR/Cas Gene Editing Technology in Major Crop Molecular Breeding and Improving 认领 引用 被引量：1
7
作者 Dao Yao Junming Zhou +4 位作者 Yashuo Wang Yuxin Li Wenge Cheng Xiaoyu Lu Huijing Liu 《Phyton-International Journal of Experimental Botany》 SCIE 2025年第6期1669-1694,共26页
Clustered regularly interspaced short palindromic repeat sequences(CRISPR)and their accompanying proteins(Cas),commonly presenting in bacteria and archaea,make up the CRISPR/Cas system.As one of the funda-mental sourc... Clustered regularly interspaced short palindromic repeat sequences(CRISPR)and their accompanying proteins(Cas),commonly presenting in bacteria and archaea,make up the CRISPR/Cas system.As one of the funda-mental sources of nutrition for humans,edible crops play a crucial role in ensuring global food security.CRISPR/Cas9 gene editing has been applied to improve many crop traits,such as increasing nitrogen utilization efficiency,creating male sterile germplasm,and regulating tiller and spikelet formation.This paper provides a comprehensive overview of the use of CRISPR/Cas gene editing technology in crop genomes,covering the targeted genes,the types of editing that take place,the mechanism of action.Finally,we also discussed the efficiency of gene editing and pointed the future direction on how to speed up crop molecular breeding,increase breeding effectiveness,and produce more new crop varieties with high qualities. 展开更多
关键词 CRISPR/Cas gene editing technology crop molecular breeding grain security
暂未订购 下载PDF
The Interface of Gene Editing with Regenerative Medicine 认领 引用 被引量：1
8
作者 Veronica E.Farag Elsie A.Devey Kam W.Leong 《Engineering》 SCIE EI CSCD 2025年第3期73-100,共28页
The potential of regenerative medicine in the clinical space is vast,given its ability to repair and replace damaged tissues,restore lost functions due to age or disease,and transform personalized therapy.Traditional ... The potential of regenerative medicine in the clinical space is vast,given its ability to repair and replace damaged tissues,restore lost functions due to age or disease,and transform personalized therapy.Traditional regenerative medicine and tissue engineering strategies have created specialized tissues using progenitor cells and various biological stimuli.To date,there are many US Food and Drug Administration(FDA)-approved regenerative medicine therapies,such as those for wound healing and orthopedic injuries.Nonetheless,these therapies face challenges,including off-target effects,a lack of precision,and failure to target the disease or injury at its origin.In search of novel,precise,and efficient alternatives,the regenerative medicine landscape is shifting towards genome engineering technologies,particularly gene editing.Clustered regularly interspaced short palindromic repeats(CRISPR)-based gene editing systems enable precise knock-ins,knockouts,transcriptional activation and repression,as well as specific base conversions.This advancement has allowed researchers to treat genetic and degenerative diseases,control cell fate for highly regulated tissue repair,and enhance tissue functions.In this review,we explore the progress and future prospects of CRISPR technologies in regenerative medicine,focusing on how gene editing has led to advanced therapeutic applications and served as a versatile research tool for understanding tissue development and disease progression. 展开更多
关键词 CRISPR/Cas9 Tissue engineering Regenerative medicine Gene editing Stem cell transplantation Disease models
暂未订购 下载PDF
Future prospects for the advancement of treatment of men with NOA: focus on gene editing, artificial sperm, stem cells, and use of imaging 认领 引用 被引量：1
9
作者 Akeem Babatunde Sikiru Manh Nguyen Truong Wael Zohdy 《Asian Journal of Andrology》 SCIE CAS CSCD 2025年第3期433-439,共7页
Nonobstructive azoospermia(NOA)affects about 60%of men with azoospermia,representing a severe form of male infertility.The current approach to manage NOA primarily involves testicular sperm retrieval methods such as c... Nonobstructive azoospermia(NOA)affects about 60%of men with azoospermia,representing a severe form of male infertility.The current approach to manage NOA primarily involves testicular sperm retrieval methods such as conventional testicular sperm extraction(c-TESE)and microdissection testicular sperm extraction(micro-TESE).While combining testicular sperm retrieval with intracytoplasmic sperm injection(ICSI)offers hope for patients,the overall sperm retrieval rate(SRR)stands at around 50%.In cases where micro-TESE fails to retrieve sperm,limited options,like donor sperm or adoption,can be problematic in certain cultural contexts.This paper delves into prospective treatments for NOA management.Gene editing technologies,particularly clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated(Cas)protein 9(CRISPR/Cas9),hold potential for correcting genetic mutations underlying testicular dysfunction.However,these technologies face challenges due to their complexity,potential off-target effects,ethical concerns,and affordability.This calls for research to address key challenges associated with NOA management within the clinical settings.This also necessitate ongoing research essential for developing more sensitive diagnostic tests,validating novel treatments,and customizing current treatment strategies for individual patients.This review concluded that the future of NOA management may entail a combination of these treatment options,tailored to each patient’s unique circumstances,providing a comprehensive approach to address NOA challenges. 展开更多
关键词 artificial sperm gene editing non-obstructive azoospermia spermatogonial stem cells
暂未订购 下载PDF
Auxin affects gene editing efficiency through regulating chromatin accessibility and plant regeneration process 认领 引用
10
作者 Zhaoyuan Lian Tao Jiang +8 位作者 Yufei Liang Wanxing Hu Huimin Peng Hanghang Zhang Haijun Gong Chunxiang You Guiluan Wang Li Liu Heqiang Huo 《Horticulture Research》 SCIE CSCD 2025年第12期182-198,共17页
Improving gene editing efficiency has been a prominent research focus with the increasing application of CRISPR/Cas9 in crop genetic enhancement.In this study,we demonstrated that increasing exogenous auxin levels dur... Improving gene editing efficiency has been a prominent research focus with the increasing application of CRISPR/Cas9 in crop genetic enhancement.In this study,we demonstrated that increasing exogenous auxin levels during in vitro tissue culture significantly enhances gene editing efficiency,leading to a higher frequency of functionally edited T0 plants.While higher auxin levels promoted callus growth,it also delayed shoot initiation and slightly decreased shoot regeneration.Subsequent RNA-Seq analysis revealed significant alterations in the expression of plant developmental regulatory genes and chromatin remodeling genes at two plant regeneration stages.Further analysis using nuclei staining and Transposase-Accessible Chromatin using sequencing showed that excessive auxin resulted in a more relaxed chromatin structure in callus cells,thus enhancing the genomic DNA accessibility to Cas9.Additionally,the prolonged growth period of dedifferentiated callus cells and the delay in shoot initiation likely provided additional time for Cas9 to exert its function,explaining the improved gene editing efficiency due to excessive auxin application.To mitigate the inhibitory effects of excessive auxin on shoot regeneration,a‘two-phase’culture strategy was developed and validated using tomatoes,in which the explants were first cultured in media containing excessive auxin to promote calli growth and gene editing,then transferred to the media with lower auxin concentrations to promote the following shoots regeneration.Overall,our research has revealed novel aspects of auxin function in gene editing,offering new insights and a theoretical basis for future studies.Furthermore,the proposed culture method could accelerate the application of gene editing across various plant species. 展开更多
关键词 functionally edited t plantswhile increasing exogenous auxin levels auxin plant regeneration vitro tissue culture chromatin accessibility gene editing crop genetic enhancementin
暂未订购 下载PDF
Ornamental plant gene editing:Past,present and future 认领 引用 被引量：2
11
作者 Jing Tang Jingxuan Ye +3 位作者 Peixue Liu Siqi Wang Fadi Chen Aiping Song 《Ornamental Plant Research》 2023年第1期155-160,共6页
With the rapid development of biotechnology,gene editing has become more widely used as a powerful tool to regulate plant traits directionally and efficiently.Here,we summarize the recent research progress in ornament... With the rapid development of biotechnology,gene editing has become more widely used as a powerful tool to regulate plant traits directionally and efficiently.Here,we summarize the recent research progress in ornamental plant gene editing,including flower type,flower color,vase life,marker genes and other traits.We also discuss the application potential of other crop gene editing methods in ornamental plants and explore the diversity and feasibility of gene editing techniques in plant breeding to promote the molecular breeding of ornamental plants. 展开更多
关键词 regulate plant traits ornamental plants gene editingincluding plant breeding gene editing biotechnologygene editing
暂未订购 下载PDF
Gene editing: an instrument for practical application of gene biology to plant breeding 认领 引用 被引量：11
12
作者 Yuan-yuan TAN Hao DU +5 位作者 Xia WU Yan-hua LIU Meng JIANG Shi-yong SONG Liang WU Qing-yao SHU 《Journal of Zhejiang University-SCIENCE B》 SCIE CAS CSCD 2020年第6期460-473,共14页
Plant breeding is well recognized as one of the most important means to meet food security challenges caused by the ever-increasing world population. During the past three decades, plant breeding has been empowered by... Plant breeding is well recognized as one of the most important means to meet food security challenges caused by the ever-increasing world population. During the past three decades, plant breeding has been empowered by both new knowledge on trait development and regulation(e.g., functional genomics) and new technologies(e.g., biotechnologies and phenomics). Gene editing, particularly by clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein(Cas) and its variants, has become a powerful technology in plant research and may become a game-changer in plant breeding. Traits are conferred by coding and non-coding genes. From this perspective, we propose different editing strategies for these two types of genes. The activity of an encoded enzyme and its quantity are regulated at transcriptional and post-transcriptional, as well as translational and post-translational, levels. Different strategies are proposed to intervene to generate gene functional variations and consequently phenotype changes. For non-coding genes, trait modification could be achieved by regulating transcription of their own or target genes via gene editing. Also included is a scheme of protoplast editing to make gene editing more applicable in plant breeding. In summary, this review provides breeders with a host of options to translate gene biology into practical breeding strategies, i.e., to use gene editing as a mechanism to commercialize gene biology in plant breeding. 展开更多
关键词 Gene editing Expression regulation Novel allele Trait development Plant breeding
暂未订购 下载PDF
Efficient generation of the mouse model with a defined point mutation through haploid cell-mediated gene editing 认领 引用 被引量：7
13
作者 Leixin Wei Xiukun Wang +2 位作者 Suming Yang Wen Yuan Jinsong Li 《Journal of Genetics and Genomics》 SCIE CAS CSCD 2017年第9期461-463,共3页
Generation of mouse models carrying a defined point mutation,especially disease-related point mutations,is of considerable interest for research in biology and medicine.The standard method based on embryonic stem cell... Generation of mouse models carrying a defined point mutation,especially disease-related point mutations,is of considerable interest for research in biology and medicine.The standard method based on embryonic stem cell（ESC）-mediated homologous recombination（HR）is time-and labor-consuming. 展开更多
关键词 ESC AG Efficient generation of the mouse model with a defined point mutation through haploid cell-mediated gene editing
暂未订购 下载PDF
Efficient gene editing in a medaka(Oryzias latipes)cell line and embryos by SpCas9/tRNA-gRNA 认领 引用 被引量：4
14
作者 Qihua PAN Junzhi LUO +3 位作者 Yuewen JIANG Zhi WANG Ke LU Tiansheng CHEN 《Journal of Zhejiang University-SCIENCE B》 SCIE CAS CSCD 2022年第1期74-83,共10页
Generation of mutants with clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9(Cas9)is commonly carried out in fish species by co-injecting a mixture of Cas9 messenger RNA(mRN... Generation of mutants with clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9(Cas9)is commonly carried out in fish species by co-injecting a mixture of Cas9 messenger RNA(mRNA)or protein and transcribed guide RNA(gRNA).However,the appropriate expression system to produce functional gRNAs in fish embryos and cells is rarely present.In this study,we employed a poly-transfer RNA(tRNA)-gRNA(PTG)system driven by cytomegalovirus(CMV)promoter to target the medaka(Oryzias latipes)endogenous gene tyrosinase(tyr)or paired box 6.1(pax6.1)and illustrated its function in a medaka cell line and embryos.The PTG system was combined with the CRISPR/Cas9 system under high levels of promoter to successfully induce gene editing in medaka.This is a valuable step forward in potential application of the CRISPR/Cas9 system in medaka and other teleosts. 展开更多
关键词 Medaka(Oryzias latipes) Gene editing Poly-tRNA-gRNA Embryos Fish cells
暂未订购 下载PDF
Gene therapy and gene editing strategies in inherited blood disorders 认领 引用 被引量：2
15
作者 Xuemei Song JinLei Liu +3 位作者 Tangcong Chen Tingfeng Zheng Xiaolong Wang Xiang Guo 《Journal of Genetics and Genomics》 SCIE CAS CSCD 2024年第11期1162-1172,共11页
Gene therapy has shown significant potential in treating various diseases,particularly inherited blood disorders such as hemophilia,sickle cell disease,and thalassemia.Advances in understanding the regulatory network ... Gene therapy has shown significant potential in treating various diseases,particularly inherited blood disorders such as hemophilia,sickle cell disease,and thalassemia.Advances in understanding the regulatory network of disease-associated genes have led to the identification of additional therapeutic targets for treatment,especially for β-hemoglobinopathies.Erythroid regulatory factor BCL11A offers the most promising therapeutic target for β-hemoglobinopathies,and reduction of its expression using the commercialized gene therapy product Casgevy has been approved for use in the UK and USA in 2023.Notably,the emergence of innovative gene editing technologies has further broadened the gene therapy landscape,presenting possibilities for treatment.Intensive studies indicate that base editing and prime editing,built upon CRISPR technology,enable precise single-base modification in hematopoietic stem cells for addressing inherited blood disorders ex vivo and in vivo.In this review,we present an overview of the current landscape of gene therapies,focusing on clinical research and gene therapy products for inherited blood disorders,evaluation of potential gene targets,and the gene editing tools employed in current gene therapy practices,which provides an insight for the establishment of safer and more effective gene therapy methods for a wider range of diseases in the future. 展开更多
关键词 Gene therapy Inherited blood disorders Therapeutic targets β-Hemoglobinopathies Gene editing
暂未订购 下载PDF
A zwitterionic polymer-inspired material mediated efficient CRISPR-Cas9 gene editing 认领 引用 被引量：1
16
作者 Lingmin Zhang Langyu Yang +7 位作者 Jionghua Huang Sheng Chen Chuangjia Huang Yinshan Lin Ao Shen ZhouYikang Zheng Wenfu Zheng Shunqing Tang 《Asian Journal of Pharmaceutical Sciences》 SCIE CAS 2022年第5期666-678,共13页
The typeⅡ prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 (CRISPR/Cas9) adaptive immune system is a cutting-edge genome-editing toolbox.However,its applications are still limited b... The typeⅡ prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 (CRISPR/Cas9) adaptive immune system is a cutting-edge genome-editing toolbox.However,its applications are still limited by its inefficient transduction.Herein,we present a novel gene vector,the zwitterionic polymer-inspired material with branched structure (ZEBRA) for efficient CRISPR/Cas9 delivery.Polo-like kinase 1 (PLK1) acts as a master regulator of mitosis and overexpresses in multiple tumor cells.The Cas9 and single guide sgRNA (sgRNA)-encoded plasmid was transduced to knockout Plk1 gene,which was expected to inhibit the expression of PLK1.Our studies demonstrated that ZEBRA enabled to transduce the CRISPR/Cas9 system with large size into the cells efficiently.The transduction with ZEBRA was cell line dependent,which showed~10-fold higher in CD44-positive cancer cell lines compared with CD44-negative ones.Furthermore,ZEBRA induced highlevel expression of Cas9 proteins by the delivery of CRISPR/Cas9 and efficient gene editing of Plk1 gene,and inhibited the tumor cell growth significantly.This zwitterionic polymerinspired material is an effective and targeted gene delivery vector and further studies are required to explore its potential in gene delivery applications. 展开更多
关键词 CRISPR/Cas9 Gene editing Zwitterionic polymers CD44 PLK1
暂未订购 下载PDF
Lipid nanoparticle-mediated CRISPR/Cas9 gene editing and metabolic engineering for anticancer immunotherapy 认领 引用 被引量：4
17
作者 Hyemin Ju Dongyoon Kim Yu-Kyoung Oh 《Asian Journal of Pharmaceutical Sciences》 SCIE CAS 2022年第5期641-652,共12页
Metabolic engineering of the tumor microenvironment has emerged as a new strategy.Lactate dehydrogenase A(LDHA)is a prominent target for metabolic engineering.Here,we designed a cationic lipid nanoparticle formulation... Metabolic engineering of the tumor microenvironment has emerged as a new strategy.Lactate dehydrogenase A(LDHA)is a prominent target for metabolic engineering.Here,we designed a cationic lipid nanoparticle formulation for LDHA gene editing.The plasmid DNA delivery efficiency of our lipid nanoparticle formulations was screened by testing the fluorescence of lipid nanoparticles complexed to plasmid DNA encoding green fluorescence protein(GFP).The delivery efficiency was affected by the ratios of three components:a cationic lipid,cholesterol or its derivative,and a fusogenic lipid.The lipid nanoparticle designated formulation F3 was complexed to plasmid DNA co-encoding CRISPR-associated protein 9 and LDHA-specific sgRNA,yielding the lipoplex,pCas9-sgLDHA/F3.The lipoplex including GFP-encoding plasmid DNA provided gene editing in HeLa-GFP cells.Treatment of B16F10 tumor cells with pCas9-sgLDHA/F3 yielded editing of the LDHA gene and increased the pH of the culture medium.pCas9-sgLDHA/F3 treatment activated the interferon-gamma and granzyme production of T cells in culture.In vivo,combining pCas9-sgLDHA/F3 with immune checkpoint-inhibiting anti-PD-L1 antibody provided a synergistic antitumor effect and prolonged the survival of tumor model mice.This study suggests that combining metabolic engineering of the tumor microenvironment with immune checkpoint inhibition could be a valuable antitumor strategy. 展开更多
关键词 Gene editing Lipid nanoparticle Metabolic engineering Lactate dehydrogenase A Tumor microenvironment
暂未订购 下载PDF
Ethical and legal implications of gene editing in plant breeding:a systematic literature review 认领 引用 被引量：3
18
作者 Siti Hafsyah IDRIS Nurzatil Sharleeza MAT JALALUDDIN Lee Wei CHANG 《Journal of Zhejiang University-SCIENCE B》 SCIE CAS CSCD 2023年第12期1093-1105,共13页
Biotechnology policies and regulations must be revised and updated to reflect the most recent advances in plantbreeding technology. New Plant Breeding Techniques(NPBT) such as gene editing have been applied to address... Biotechnology policies and regulations must be revised and updated to reflect the most recent advances in plantbreeding technology. New Plant Breeding Techniques(NPBT) such as gene editing have been applied to address the myriad of challenges in plant breeding, while the use of NPBT as emerging biotechnological tools raises legal and ethical concerns. This study aims to highlight how gene editing is operationalized in the existing literature and examine the critical issues of ethical and legal issues of gene editing for plant breeding. We carried out a systematic literature review(SLR) to provide the current states of ethical and legal discourses surrounding this topic. We also identified critical research priority areas and policy gaps that must be addressed when designing the future governance of gene editing in plant breeding. 展开更多
关键词 Systematic literature review Gene editing Plant breeding Ethical and legal issues Consequentialism Virtue ethics principles
暂未订购 下载PDF
Updates on CRISPR-based gene editing in HIV-1/AIDS therapy 认领 引用 被引量：3
19
作者 Zhihao Zhang Wei Hou Shuliang Chen 《Virologica Sinica》 SCIE CAS CSCD 2022年第1期1-10,共10页
Although tremendous efforts have been made to prevent and treat HIV-1 infection,HIV-1/AIDS remains a major threat to global human health.The combination antiretroviral therapy(cART),although able to suppress HIV-1 rep... Although tremendous efforts have been made to prevent and treat HIV-1 infection,HIV-1/AIDS remains a major threat to global human health.The combination antiretroviral therapy(cART),although able to suppress HIV-1 replication,cannot eliminate the proviral DNA integrated into the human genome and thus requires lifelong treatment that may lead to various side effects.In recent years,clustered regularly interspaced short palindromic repeat(CRISPR)-associated nuclease 9(Cas9)related gene-editing systems have been developed and designed as effective ways to treat HIV-1 infection.However,new gene-targeting tools derived from or functioning like CRISPR/Cas9,including base editor,prime editing,SHERLOCK,DETECTR,PAC-MAN,ABACAS,pfAGO,have been developed and optimized for pathogens detection and diseases correction.Here,we summarize recent studies on HIV-1/AIDS gene therapy and provide more gene-editing targets based on studies relating to the molecular mechanism of HIV-1 infection.We also identify the strategies and potential applications of these new gene-editing technologies for HIV-1/AIDS treatment in the future.Moreover,we discuss the caveats and problems that should be addressed before the clinical use of these versatile CRISPR-based gene targeting tools.Finally,we offer alternative solutions to improve the practice of gene targeting in HIV-1/AIDS gene therapy. 展开更多
关键词 HIV-1/AIDS CRISPR/Cas9 Gene editing Gene therapy
暂未订购 下载PDF
Therapeutic gene editing strategies using CRISPR-Cas9 for theβ-hemoglobinopathies 认领 引用 被引量：1
20
作者 James B.Papizan Shaina N.Porter +1 位作者 Akshay Sharma Shondra M.Pruett-Miller 《Journal of Biomedical Research》 CAS CSCD 2021年第2期115-134,共20页
With advancements in gene editing technologies,our ability to make precise and efficient modifications to the genome is increasing at a remarkable rate,paving the way for scientists and clinicians to uniquely treat a ... With advancements in gene editing technologies,our ability to make precise and efficient modifications to the genome is increasing at a remarkable rate,paving the way for scientists and clinicians to uniquely treat a multitude of previously irremediable diseases.CRISPR-Cas9,short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9,is a gene editing platform with the ability to alter the nucleotide sequence of the genome in living cells.This technology is increasing the number and pace at which new gene editing treatments for genetic disorders are moving toward the clinic.Theβ-hemoglobinopathies are a group of monogenic diseases,which despite their high prevalence and chronic debilitating nature,continue to have few therapeutic options available.In this review,we will discuss our existing comprehension of the genetics and current state of treatment forβ-hemoglobinopathies,consider potential genome editing therapeutic strategies,and provide an overview of the current state of clinical trials using CRISPR-Cas9 gene editing. 展开更多
关键词 sickle cell disease sickle cell anemia fetal hemoglobin hemoglobinopathy CRISPR gene editing genome engineering
暂未订购 下载PDF
已选0条
引用分析
参考文献
引证文献
 统计分析
检索结果
已选文献
上一页 1 2 … 13 下一页 到第 页
在线咨询 使用帮助 返回顶部 意见反馈