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• Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative Medicine.
  Background: β-haemoglobinopathies, including sickle cell disease and transfusion-dependent β-thalassaemia, are among the most common monogenic disorders worldwide and represent a major global health burden. Conventional treatments, such as blood tr…
  pubmed Fogliazza F, Carbone G, Berzieri M, Ciriaco D 等 2026 Jun 26 置信度 0.82
• Application of CRISPR-Cas9-Based Gene Editing Technology in Inherited Liver Diseases.
  Inherited liver diseases are predominantly caused by monogenic mutations, and the vast majority of these conditions currently lack curative treatment options. Although liver transplantation may be used for patients with end-stage disease, it faces numerous cha…
  pubmed Liu R, Cong S, Gao Y, Xu J 等 2026 Jul 21 置信度 0.82
• Beyond Permanent Genome Editing: Molecular Delivery Strategies for RNA Editing and Epigenome-Editing Therapeutics.
  Reversible genetic medicines are emerging as controllable alternatives to permanent genome editing by enabling programmable modulation of RNA sequence, transcript abundance, chromatin state, and gene expression without irreversible genomic alteration. However,…
  pubmed Zaman W, Ayaz A 2026 Jul 21 置信度 0.82
• Efficient Gene Editing in Fish Primary Germline Stem Cells.
  Genome editing by the CRISPR/Cas9 system is widely used for production of gene-modified animals, including fish. However, efficient gene editing in fish cultured cells, in particular germline stem cells (GSCs), is challenging, likely due to the difficulty in t…
  pubmed Zhan Y, Luo T, Sun Y 2026 Jul 15 置信度 0.82
• Plant Regeneration: Influencing Factors, Regulatory Networks, and Epigenetic Mechanisms.
  Plant tissue culture is a crucial part of biotechnology that supports crop improvement, plant conservation, and other related fields. Although tissue culture has been successfully used in many plants, low regeneration and transformation rates still exist in so…
  pubmed Song W, Cheng X, Liu X, Wang L 等 2026 Jul 14 置信度 0.82
• Pipettes and Pipelines: The Weapons of Omics Sciences for a New Age of Clinical Studies.
  The omics sciences represent a revolution for clinical studies, offering integrative approaches to analyzing biological data with unprecedented depth. From the discovery of the double-helix structure of DNA to the CRISPR-Cas9 gene editing tool, passing through…
  pubmed Lopes ÍS, Fukutani ER, Mota TF, Andrade BB 等 2026 Jul 18 置信度 0.82
• Research Progress on the Regulatory Mechanisms of Salt-Stress Response and Functional Genes in Populus.
  Soil salinization represents one of the most severe abiotic constraints on global forest productivity. Populus , the most widely cultivated fast-growing timber tree and a premier model woody plant, exhibits striking intrageneric variation in salt tolerance-fro…
  pubmed He P, Cai H 2026 Jul 3 置信度 0.82
• Gene-Edited Stem Cells for Ischemic Vascular Disease: Current Advances and Future Perspectives.
  Ischemic vascular diseases remain a leading cause of morbidity and mortality worldwide and are frequently associated with irreversible tissue damage. Although stem cell-based therapies have shown promise for vascular regeneration, their clinical translation ha…
  pubmed Han S, Kim SW 2026 Jul 2 置信度 0.82
• The Role of Long Non-Coding RNAs in the Pathogenesis of Coronary Heart Disease.
  Long non-coding RNAs (lncRNAs) constitute an important group of regulatory RNA molecules involved in the control of gene expression at the epigenetic, transcriptional, and post-transcriptional levels. In recent years, their crucial role in the pathophysiology …
  pubmed Plewa P, Kulpa J, Szulc J, Szczepanik M 等 2026 Jul 15 置信度 0.82
• Oligonucleotide Synthesis Errors Are a Source of Untoward Variation in HDR-Mediated Gene Editing.
  Background/Objectives: Single-stranded oligonucleotides (ssODNs) are used as donor templates for therapeutic gene editing by CRISPR-Cas9 cleavage and homology-directed repair (HDR). Although ssODN sequence fidelity is critical to the safety and efficacy of edi…
  pubmed Wyman SK, Romero Z, Heo SJ, Navarrete M 等 2026 Jun 24 置信度 0.82
• Redox Regulation of Plant-Root-Knot Nematode Interactions: From ROS-Mediated Immunity to Sustainable Resistance.
  Root-knot nematodes (RKNs; Meloidogyne spp.) are among the most destructive plant parasites, causing severe yield losses in diverse crops. Reactive oxygen species (ROS), particularly superoxide radicals (O 2 • - ) and hydrogen peroxide (H 2 O 2 ), are c…
  pubmed Yang JW, Kim HS, Kim YH 2026 Jul 6 置信度 0.82
• Restoring Cancer Genomes: Functional Mutation Correction as a Platform for Precision Oncology.
  Cancer genome sequencing has uncovered an extensive landscape of somatic mutations. However, determining which of these alterations are biologically consequential and therapeutically actionable remains a central challenge in oncology. CRISPR-based genome editi…
  pubmed Wang P, Sayed S, Buchholz F 2026 Jul 27 置信度 0.82
• An abundant merozoite surface protein of Plasmodium falciparum modulates susceptibility to inhibitory antibodies.
  Malaria merozoite surface proteins (MSPs) are thought to have important roles in red blood cell (RBC) invasion and their exposure on the parasite surface makes them attractive vaccine candidates. However, their role in invasion has not been directly demonstrat…
  pubmed Henshall IG, Chmielewski J, Angage D, Romeo O 等 2026 Jul 27 置信度 0.82
• Lonvoguran ziclumeran: a CRISPR-CAS9-based gene therapy for the treatment of hereditary angioedema.
  Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent swelling caused by dysregulation of the kallikrein-kinin pathway. Although current therapies effectively reduce attack frequency, treatment remains lifelong. Lonvoguran ziclumera…
  pubmed Luong LH, Stone S, Bui V, Bhattarai B 等 2026 Jul 29 置信度 0.82
• [Advances in research on the molecular mechanisms and gene therapy of hereditary hearing impairment].
  Hereditary hearing impairment represents a significant etiology of language and social dysfunction in both children and adults, primarily caused by genetic factors. To date, over 150 genes have been identified in association with this disorder. The pathogenic …
  pubmed Yang XP, Gao YQ, Huang L, Zhao LJ 等 2026 Jul 6 置信度 0.82
• Systematic Benchmarking of CRISPR-Cas9 Off-Target Prediction Tools Reveals Limitations and Implications for Preclinical Assessment.
  Accurate identification of CRISPR-Cas9 off-target sites is essential for the safety assessment of genome-editing-based therapies. While numerous in silico prediction tools have been developed, their comparative performance and practical utility in preclinical …
  pubmed Kaufmann MM, Hackenberg M, Jobson Pargeter W, Backofen R 等 2026 Jul 26 置信度 0.82
• The CCCH-type zinc finger protein SZF12 negatively regulates salt stress tolerance in rice by suppressing stress-responsive transcriptional reprogramming.
  Soil salinity represents one of the most damaging abiotic stresses constraining rice (Oryza sativa L.) production globally. Although CCCH-type zinc finger proteins have emerged as important regulators of stress responses in plants, the molecular mechanisms by …
  pubmed Lei H, Zhu Y, Du Q, Jiang L 等 2026 Jul 23 置信度 0.82
• Manipulating insect sex is a promising pest control strategy: from theory to application.
  Insect sex manipulation has emerged as a transformative approach in sustainable pest management. This review summarizes the molecular mechanisms underlying insect sex determination and their application in artificial sex manipulation, offering novel strategies…
  pubmed Huang M, Gong RN, Smagghe G, Zang LS 等 2026 Jul 26 置信度 0.82
• In utero gene editing and precision medicine.
  Advances in preconception and prenatal genetic testing, in combination with the rapid evolution and regulatory acceptance of targeted gene therapy technologies, has unveiled promising opportunities for the antenatal treatment of genetic disease by way of in ut…
  pubmed Katz OL, Kennedy K, Berger NF, Fadl N 等 2026 Jul 16 置信度 0.82
• Retinal Pigment Epithelium and Microglia Transplantation in Age-related Macular Degeneration.
  Age-related macular degeneration (AMD) remains a leading cause of irreversible blindness worldwide, characterized by the progressive breakdown of the outer blood-retinal barrier, accumulation of protein and lipid deposits in the subretinal space, and the conse…
  pubmed Surawatsatien N, Solanky RM, van de Werken RP, Kong M 等 2026 Jul 25 置信度 0.82
• Multi-copy expression of classical swine fever virus E2 in a recombinant pseudorabies virus enhances immunogenicity and supports its potential as a PRV-CSFV bivalent vaccine candidate.
  Classical swine fever virus (CSFV) and pseudorabies virus (PRV) remain significant threats to the swine industry. Although recombinant PRV vectors represent promising platforms for bivalent vaccine development, their application is frequently limited by insuff…
  pubmed Shang X, Zhao H, He Y, Rong Z 等 2026 Jul 22 置信度 0.82
• Optimization of genome editing strategies for targeted integration at chicken sex chromosome loci.
  The precise targeted integration of large exogenous genes into chicken sex chromosomes is of particular interest for sex-linked trait manipulation, sex-control breeding, and the development of avian bioreactor models. However, efficient targeted integration at…
  pubmed Cao Z, Chen C, He Y, Wei W 等 2026 Jul 5 置信度 0.82
• DAZL-targeted inducible kill switches enable efficient ablation of chicken primordial germ cells.
  Competition from endogenous primordial germ cells (PGCs) in recipient embryos limits the generation of fully donor-derived offspring in avian surrogate-host systems. An inducible and germline-restricted ablation strategy is therefore needed to create sterile r…
  pubmed Qin C, Jiang S, Xiao F, Huang J 等 2026 Jul 17 置信度 0.82
• Gene therapy approaches for inborn errors of immunity: from bench to bedside.
  Inborn errors of immunity (IEI) are rare genetic defects that disrupt immune function, often resulting in life-threatening infections, malignancies, and immune dysregulation. Allogeneic hematopoietic stem cell transplantation (HSCT), a curative option for some…
  pubmed Dara J, Booth C 2026 Jul 25 置信度 0.82
• Phospholipase C-Delta1 Deletion Impairs Dermal Papilla Cell Function by Disrupting the HIF-1/MAPK Signaling Pathway.
  Phospholipase c-delta 1 (PLCD1), a key effector in the phospholipid signaling pathway, is indispensable for the growth and development of hair follicles (HF), but its regulatory mechanism in dermal papilla cells (DPCs), the core functional cells of HF, remains…
  pubmed Cai J, Zhao B, Li Y, Chen X 等 2026 Jul 31 置信度 0.82
• Beyond GMOs: transgene-free gene-edited crops for global food security.
  Transgene-free gene-editing has transformed the genomic landscape of crops by enabling targeted, precise, and predictable genetic outcomes without integrating any foreign DNA into the host genome. It has significantly reduced production time and costs, and the…
  pubmed Ahmad A, Faheem M, Ijaz A, Niamat A 等 2026 置信度 0.82
• Wildlife Trade and Genetic Basis of Disease Susceptibility: A Review.
  The surge in the trade of wildlife and wildlife products drives several species to extinction while coinciding with the increase in several zoonotic diseases. It is therefore essential to explore the roles of wildlife trade in disease transmission, and how the…
  pubmed Coker OM, Fagbenro OS, Coker MM 2026 Jul 25 置信度 0.82
• The novel transcriptional activator Bhr1 combining NTPase and Zn(II)2Cys6 DNA-binding domains controls (hemi-)cellulase response to mannose-rich substrates in the white-rot fungus Dichomitus squalens.
  The regulatory landscape responsible for lignocellulose degradation in white-rot basidiomycete fungi remains largely unexplored. In this study, we characterize a novel transcriptional activator, Bhr1, in the white-rot fungus Dichomitus squalens . Bhr1 exhibits…
  pubmed Gonzalez Ramos VM, Lipzen A, Hundley H, Ng V 等 2026 Jul 24 置信度 0.82
• Development of potato (Solanum tuberosum L.) plants with inactivated StPain-1 gene using CRISPR/Cas9.
  Storage of potato tubers intended for further processing is complicated by their cold-induced sweetening (CIS). Enzymatic hydrolysis of sucrose into glucose and fructose occurs at low temperatures. The resulting high hexose content adversely affects the qualit…
  pubmed Karlov VD, Volkov MK, Antipov AD, Monahova YS 等 2026 Jul 置信度 0.82
• CRISPR/Cas9-mediated knockout of MsMLO1 confers enhanced powdery mildew resistance in alfalfa.
  Powdery mildew, a disease primarily caused by Erysiphe pisi , is one of the most devastating foliar diseases of alfalfa ( Medicago sativa L.), a globally important forage crop. The MLO gene is a well-known susceptibility factor for powdery mildew in many plant…
  pubmed Hou Z, Li Q, Wang Y, Zong H 等 2026 Dec 置信度 0.82
• Selection of two pronuclei zygotes before electroporation does not improve gene editing efficiency or reduce mosaicism in porcine embryos despite optimized timing and Hoechst staining.
  Electroporation-mediated delivery of Clustered-Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 components is an effective approach for genome editing in porcine embryos; however, mosaicism remains a major limitation. Select…
  pubmed Takebayashi K, Otoi T, Wittayarat M, Setyo Widodo O 等 2026 Jun 置信度 0.82
• VvERF105 enhances drought resistance in grape through interaction with VvSnRK1.
  Drought stress severely restricts grape growth and yield. ERF105 is widely involved in plant developmental processes as well as responses to biotic and abiotic stresses. Nevertheless, existing studies of the ERF105 gene have primarily focused on cold and disea…
  pubmed Cui X, Lou Y, Zhang K, Lu H 等 2026 置信度 0.82
• CRISPRing through time: How cutting-edge technology is revolutionizing life sciences and medicine.
  Given the plethora of emerging technologies, none have truly captured the minds as CRISPR. From the groundbreaking research, the ultimate battle of the prizes and patents to a number of books, the science of CRISPR continues to be significant in the biomedical…
  pubmed Banik I, Coppé JP 2026 Sep 8 置信度 0.82
• Engineering climate-resilient horticultural crops: advances in transcriptional regulation, genome editing, and synthetic networks.
  Abiotic stresses-particularly cold, drought, and salinity-pose significant threats to the productivity and sustainability of horticultural crops. Recent studies have revealed conserved and species-specific regulatory mechanisms that allow plants to adapt dynam…
  pubmed Wu W, Wang R, Li J, Zhang Z 等 2026 Aug 置信度 0.82
• Boosting cytosine base editing in potato through synergistic optimization.
  Base editing enables precise substitution of single nucleotides and is a promising technology for improvement of agronomic traits, including those of potato ( Solanum tuberosum ), the third most important food crop globally. Here, we developed efficient cytosi…
  pubmed Zhang Y, Zhang L, Deng J, Zhou H 等 2026 Aug 置信度 0.82
• WSB1 is dispensable for mouse spermatogenesis and male fertility.
  WSB1 (WD repeat and SOCS box-containing protein 1) is part of the E3 ligase complex Elongin B/C-Cullin 2/5-SOCS box E3 ubiquitin ligase (ESC) complex. Our previous studies and those of others have confirmed that some SOCS box proteins, including ASB1 and ASB9,…
  pubmed Qu W, Wang X, Liu S, Zhou N 等 2026 置信度 0.82
• The Diversifying Distribution Trends of Maturing CRISPR Technologies by Addgene.
  Since the advent of Cas9-based CRISPR technologies in 2012, there has been a remarkable growth in genome editing research, literature, applications, and translational impact. Much of this research has been fueled by the global dissemination of CRISPR plasmids …
  pubmed Shepard A, Minones-Moyano E, Mork C, Pyhtila B 等 2026 Aug 置信度 0.82
• Kidney organoids as models for hereditary kidney diseases: Toward precision medicine (Review).
  Kidney organoids are important tools for modeling human development and disease, especially in chronic kidney disease (CKD), which is a global health challenge. Current treatment strategies focus on delaying disease progression by managing underlying causes, a…
  pubmed Cui S, Chen T, Zou Y, Li M 等 2026 Sep 置信度 0.82
• CRISPR/Cas9-Engineered HEK293T Cellular Model Harboring the Pathogenic CDKL5 c.172A>T Variant Recapitulates Core Molecular Phenotypes of CDKL5 Deficiency Disorder.
  Mutations in the CDKL5 gene are strongly associated with severe neurodevelopmental disorders, including atypical Rett syndrome and autism spectrum disorder (ASD).
  pubmed Faghihi F, Amani J, Gholami M, Salahshourifar I 2026 Jan-Dec 置信度 0.82
• Evaluation of a quintuple-gene-deleted PRV vector expressing PEDV S1: safety and immunogenicity in rabbits, mice, and piglets.
  Pseudorabies virus (PRV) and Porcine epidemic diarrhea virus (PEDV) are currently co-circulation among swine herd with multiple variant strains, which cause severe economic losses to the global swine industry. In the present study, a recombinant PRV-based vacc…
  pubmed Li J, Jiang J, Guo R, Wang X 等 2026 置信度 0.82
• Development and Characterization of RNA Aptamer-Mediated Modular Base Editors Containing Staphylococcus aureus Cas9 Derivatives and Novel Deaminase Orthologs.
  Base editing enables precise genome modifications without introducing DNA double-strand breaks. Using Streptococcus pyogenes Cas9 as a prototype, we previously developed a modular base editing platform in which the deaminase is recruited by an RNA aptamer engi…
  pubmed Collantes JC, Xu K, Ruiz-Urigüen M, Samaniego-Villacis AC 等 2026 Aug 置信度 0.82
• Generation and characterization of humanized CD4 knock-in mice expressing chimeric mouse/human CD4 protein.
  Humanized mouse models have become indispensable tools for investigating human gene function and disease modeling. However, conventional transgenic approaches carry the risk of unforeseen biological consequences. Here, to address this concern, we developed a n…
  pubmed Kam KM, Shiu TE, Hsieh CM, Lu WT 等 2026 Jul 22 置信度 0.82
• CRISPR gene editing - safe or unsafe?
  crossref Aparna Ayyar 2019-07-31T05:35:39Z 置信度 0.70
• Treating Chromosomal Abnormalities with CRISPR Gene Editing
  crossref Eric Japson 2025-11-19T15:08:48Z 置信度 0.70
• Gene Editing by CRISPR-Cas
  crossref Aarif Ali, Farheen Shafique 2025-07-04T10:41:22Z 置信度 0.70
• Developing Bioluminescent Forests Using CRISPR-Cas9 Gene Editing
  crossref SAHAJ SATANI 2023-11-24T18:53:51Z 置信度 0.70
• Patenting dynamics in CRISPR gene editing technologies
  crossref Prabuddha Ganguli 2021-03-07T16:49:07Z 置信度 0.70
• Making Antibiotic-Resistant E. coli Non-Resistant with CRISPR/Cas9 Gene Editing
  crossref Ilayda Cavrar 2023-12-16T10:26:35Z 置信度 0.70
• Antimicrobial resistance (AMR) management using CRISPR-Cas based genome editing
  crossref Debmitra Sen, Poulami Mukhopadhyay 2024-01-09T17:03:19Z 置信度 0.70
• CRISPRing or RISKing? Dangers Arising from Gene Editing with CRISPR-Cas9
  crossref Antonis Giakountis 2018-10-13T07:40:06Z 置信度 0.70
• Peer Review #2 of CRISPR-Analytics (CRISPR-A): a platform for precise analytics and simulations for gene editing
  crossref 2023-01-15T00:23:07Z 置信度 0.70
• Supplementary file 2. crRNA targeting sequences used for CRISPR/Cas9 gene editing.
  crossref 2019-07-09T06:00:28Z 置信度 0.70
• CRISPR Gene Editing to Treat Alcoholism and Nicotine Addiction
  crossref Divya Gill 2024-02-03T19:47:47Z 置信度 0.70
• Peer Review #1 of CRISPR-Analytics (CRISPR-A): a platform for precise analytics and simulations for gene editing
  crossref 2023-01-15T00:23:07Z 置信度 0.70
• Genomics
  This chapter displays genomics analysis with an emphasis on next-generation sequencing (NGS), and annotation of bacterial genomes. It uncovers the underlying principles of dideoxy sequencing and selected next-generation sequencing technologies. The chapter als…
  crossref Jamil Momand, Alison Mccurdy, Silvia Heubach, Nancy Warter-Perez 2023-09-15T12:26:13Z 置信度 0.70
• ISRN Genomics
  crossref 2017-12-10T11:53:43Z 置信度 0.70
• Medicinal plant genomics
  Abstract Recent developments in plant genomics have enabled a comprehensive analysis of the medicinal potential of plants based on their gene repertoire. Genes of biosynthesis pathways can be discovered through comparative genomics and through integration of t…
  crossref Christian Siadjeu, Boas Pucker 2023-08-01T07:03:18Z 置信度 0.70
• Comparative Genomics
  crossref 2007-04-27T15:47:53Z 置信度 0.70
• CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
  BACKGROUND: . METHODS: After conducting preclinical in vitro and in vivo studies, we evaluated the safety and pharmacodynamic effects of single escalating doses of NTLA-2001 in six patients with hereditary ATTR amyloidosis with polyneuropathy, three in each of…
  openalex Julian D. Gillmore, Ed Gane, Jörg Täubel, Justin Kao 等 2021-06-26 置信度 0.72 TransthyretinCRISPRGenome editingCas9Amyloidosis
• CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
  Transfusion-dependent -thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses -globin expression and fetal hemoglobin in erythro…
  openalex Haydar Frangoul, David Altshuler, Maria Domenica Cappellini, Yi-Shan Chen 等 2020-12-05 置信度 0.72 CRISPRThalassemiaGenome editingDiseaseCas9
• CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
  Genome editing tools such as the clustered regularly interspaced short palindromic repeat (CRISPR)-associated system (Cas) have been widely used to modify genes in model systems including animal zygotes and human cells, and hold tremendous promise for both bas…
  openalex Puping Liang, Yanwen Xu, Xiya Zhang, Chenhui Ding 等 2015-04-17 置信度 0.72 CRISPRGenome editingCas9BiologyHomologous recombination
• Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers
  openalex Isaac B. Hilton, Anthony D’Ippolito, Christopher M. Vockley, Pratiksha I. Thakore 等 2015-04-06 置信度 0.72 EnhancerBiologyAcetyltransferaseCRISPRPromoter
• Cas9 immunity creates challenges for CRISPR gene editing therapies
  Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 is a genome-editing technology1,2 that utilizes archaeal and bacterial Cas9 nucleases to introduce double-stranded breaks in DNA at targeted sites. These breaks can be used to remove, repl…
  europepmc Julie M. Crudele, Jeffrey S. Chamberlain 2018 置信度 0.80 CRISPRGenome editingCas9GeneGenetic enhancement
• Multiplex gene editing by CRISPR–Cpf1 using a single crRNA array
  openalex Bernd Zetsche, Matthias Heidenreich, Prarthana Mohanraju, Iana Fedorova 等 2016-12-05 置信度 0.72 Trans-activating crRNACRISPRMultiplexGenome editingComputational biology
• Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells
  openalex Van Trung Chu, Timm Weber, Benedikt Wefers, Wolfgang Wurst 等 2015-03-24 置信度 0.72 CRISPRGenome editingHomology (biology)GeneticsGene
• Off-target effects in CRISPR/Cas9 gene editing
  Gene editing stands for the methods to precisely make changes to a specific nucleic acid sequence. With the recent development of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system, gene editing has become efficient, convenient …
  openalex Congting Guo, Xiaoteng Ma, Fei Gao, Yuxuan Guo 2023-03-09 置信度 0.72 CRISPRGenome editingCas9Computational biologyGene
• CRISPR/Cas9 gene editing
  crossref 2015-07-10T19:02:00Z 置信度 0.70
• Sweeter tomatoes through CRISPR gene editing
  crossref 2024-12-20T20:15:17Z 置信度 0.70
• Expanded range for CRISPR gene editing
  crossref 2018-04-30T20:55:23Z 置信度 0.70
• CRISPR-Cas9: Gene Editing
  crossref Skylar Paul Strickland 2024-05-18T07:35:43Z 置信度 0.70
• CRISPR/Cas-Mediated Multiplex Gene Editing in Tomato (Solanum Lycopersicum L.)
  crossref Sonia Sethi 2024-03-18T08:02:35Z 置信度 0.70
• Crispr Gene Editing for Secondary Metabolite Production: A Review
  crossref Ali Asger Bhojiya, Harshada Joshi 2024-03-18T04:02:35Z 置信度 0.70
• The CRISPR Code: Gene Editing, Biotechnology, and the Future of Humanity
  Abstract: The CRISPR revolution has ushered in a new era of gene editing, synthetic biology, and precision medicine, allowing humanity to rewrite the genetic blueprint of life. This chapter explores the profound impact of CRISPR-Cas9 and next-generation gene-e…
  crossref Murali Krishna Pasupuleti 2025-02-21T13:36:07Z 置信度 0.70
• Gene Editing of Saccharomyces Cerevisiae Using CRISPR
  crossref Chenyu Yang, Yilin Li 2023-09-19T17:49:11Z 置信度 0.70
• CRISPR/Cas9 gene editing in hiPSC v1
  This protocol is adapted from Skarnes et al., 2019 and describes the steps to follow for knocking-in point mutation via CRISP/Cas9 in humain inducible pluripotent cells (hiPSC)
  crossref Laura Mahoney 2025-02-21T16:21:03Z 置信度 0.70
• Fanzor: A Promising CRISPR-like Gene Editing Tool in Eukaryotes
  crossref Fatih Kocabas 2023-12-16T10:58:10Z 置信度 0.70
• Gene therapy and gene editing in human disease: CRISPR and beyond
  crossref Sarah Sabin 2023-09-23T20:19:05Z 置信度 0.70
• Progress and problems with the use of viral vectors for gene therapy
  openalex Clare E. Thomas, Anja Ehrhardt, Mark A. Kay 2003-05-01 置信度 0.72 Genetic enhancementDiseaseBiologyViral vectorIntensive care medicine
• The Basic Science of Gene Therapy
  The development over the past decade of methods for delivering genes to mammalian cells has stimulated great interest in the possibility of treating human disease by gene-based therapies. However, despite substantial progress, a number of key technical issues …
  openalex Richard C. Mulligan 1993-05-14 置信度 0.72 Genetic enhancementCell therapyGene deliveryTransplantationGene
• Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease
  Severe combined immunodeficiency-X1 (SCID-X1) is an X-linked inherited disorder characterized by an early block in T and natural killer (NK) lymphocyte differentiation. This block is caused by mutations of the gene encoding the gammac cytokine receptor subunit…
  openalex Marina Cavazzana, Salima Hacein‐Bey, Geneviève de Saint Basile, Fabian Gross 等 2000-04-28 置信度 0.72 Genetic enhancementSevere combined immunodeficiencyBiologyImmunologyCommon gamma chain
• Adeno-associated virus vector as a platform for gene therapy delivery
  openalex Dan Wang, Phillip W.L. Tai, Guangping Gao 2019-02-01 置信度 0.72 Genetic enhancementAdeno-associated virusGene deliveryVector (molecular biology)Virology
• Gene therapy - promises, problems and prospects
  openalex Inder M. Verma, Nikunj V. Somia 1997-09-18 置信度 0.72 Genetic enhancementIntensive care medicineDiseaseVectors in gene therapyGene delivery
• Gene therapy comes of age
  After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, …
  openalex Cynthia E. Dunbar, Katherine A. High, J. Keith Joung, Donald B. Kohn 等 2018-01-12 置信度 0.72 Genetic enhancementGene transferBlindnessMedicineAdverse effect
• <i>LMO2</i> -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
  We have previously shown correction of X-linked severe combined immunodeficiency [SCID-X1, also known as gamma chain (gamma(c)) deficiency] in 9 out of 10 patients by retrovirus-mediated gamma(c) gene transfer into autologous CD34 bone marrow cells. However, a…
  openalex S. Hacein-Bey-Abina, Christof von Kalle, Manfred Schmidt, Matthew P. McCormack 等 2003-10-16 置信度 0.72 Genetic enhancementGeneBiologyCell growthGenetics
• Gene therapy returns to centre stage
  openalex Luigi Naldini 2015-10-01 置信度 0.72 Genetic enhancementGeneGenome editingClinical trialMedicine
• Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
  There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells, and has…
  openalex Michael Naso, Brian Tomkowicz, William L. Perry, William R. Strohl 2017-07-01 置信度 0.72 Adeno-associated virusGenetic enhancementGene deliveryVector (molecular biology)Virus
• Viral vector platforms within the gene therapy landscape
  Throughout its 40-year history, the field of gene therapy has been marked by many transitions. It has seen great strides in combating human disease, has given hope to patients and families with limited treatment options, but has also been subject to many setba…
  openalex Jote Bulcha, Yi Wang, Hong Ma, Phillip W.L. Tai 等 2021-02-08 置信度 0.72 Vector (molecular biology)ModalitiesGenetic enhancementViral vectorMedicine
• Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
  Early-onset, severe retinal dystrophy caused by mutations in the gene encoding retinal pigment epithelium-specific 65-kD protein (RPE65) is associated with poor vision at birth and complete loss of vision in early adulthood. We administered to three young adul…
  openalex James Bainbridge, Alexander J. Smith, Susie S. Barker, Scott Robbie 等 2008-04-27 置信度 0.72 MedicineRPE65Cis-trans-IsomerasesMicroperimetryVisual acuity
• Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
  Previously, several individuals with X-linked SCID (SCID-X1) were treated by gene therapy to restore the missing IL-2 receptor gamma (IL2RG) gene to CD34+ BM precursor cells using gammaretroviral vectors. While 9 of 10 patients were successfully treated, 4 of …
  openalex Salima Hacein‐Bey‐Abina, Alexandrine Garrigue, Gary P. Wang, Jean Soulier 等 2008-08-06 置信度 0.72 Genetic enhancementCancer researchInsertional mutagenesisSevere combined immunodeficiencyLeukemia
• Gene therapy in the clinic: human trials of gene therapy
  crossref Thomas Valere 2023-05-04T06:22:01Z 置信度 0.70
• Guidance for Human Somatic Cell Therapy and Gene Therapy
  crossref 2002-07-26T18:11:36Z 置信度 0.70
• Gene Therapy Briefs
  crossref 2013-03-20T14:56:34Z 置信度 0.70
• Cell and Gene Therapy World
  crossref 2016-03-23T11:47:17Z 置信度 0.70
• Gene targeting as an approach to gene therapy
  crossref Andrew Porter 2020-09-04T12:59:24Z 置信度 0.70
• Gene therapy Progress and Prospects: Gene therapy for the hemophilias
  crossref Christopher E Walsh 2003-05-30T10:49:17Z 置信度 0.70
• Gene Therapy Briefs
  crossref Alex Philippidis 2012-02-10T13:28:16Z 置信度 0.70
• Gene Therapy Progress and Prospects: Gene therapy for diabetes mellitus
  crossref V Yechoor, L Chan 2004-10-21T09:20:31Z 置信度 0.70
• DNA sequencing with chain-terminating inhibitors
  A new method for determining nucleotide sequences in DNA is described. It is similar to the "plus and minus" method [Sanger, F. & Coulson, A. R. (1975) J. Mol. Biol. 94, 441-448] but makes use of the 2',3'-dideoxy and arabinonucleoside analogues of the normal …
  openalex Frederick Sanger, S. Nicklen, Alan Coulson 1977-12-01 置信度 0.72 DNASanger sequencingNucleotideDNA polymerasePolymerase chain reaction
• The Genome Analysis Toolkit: A MapReduce framework for analyzing next-generation DNA sequencing data
  Next-generation DNA sequencing (NGS) projects, such as the 1000 Genomes Project, are already revolutionizing our understanding of genetic variation among individuals. However, the massive data sets generated by NGS--the 1000 Genome pilot alone includes nearly …
  openalex Aaron McKenna, Matthew G. Hanna, Eric Banks, Andrey Sivachenko 等 2010-07-19 置信度 0.72 Computer scienceGenomeCorrectness1000 Genomes ProjectSet (abstract data type)
• A framework for variation discovery and genotyping using next-generation DNA sequencing data
  openalex Mark A. DePristo, Eric Banks, Ryan Poplin, Kiran Garimella 等 2011-04-10 置信度 0.72 BiologyIndelGenotypingDNA sequencingExome sequencing