Highly efficient endogenous human gene correction using designed zinc-finger nucleases

No abstract is available for this paper.

• Publication year

  2005

• Venue

  Nature

• Publication date

  2005-06-02

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.1038/nature03556PMID 15806097
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Pointing fingers at the limiting step in gene targeting

  2003cited by this paper
• Zinc-finger protein-targeted gene regulation: Genomewide single-gene specificity

  2003cited by this paper
• Molecular views of recombination proteins and their control

  2003cited by this paper
• Drug discovery with engineered zinc-finger proteins

  2003cited by this paper
• Enhancing Gene Targeting with Designed Zinc Finger Nucleases

  2003cited by this paper
• Chimeric Nucleases Stimulate Gene Targeting in Human Cells

  2003cited by this paper
• Occurrence of leukaemia following gene therapy of X-linked SCID

  2003cited by this paper
• Gene therapy for the hemoglobin disorders.

  2003cited by this paper
• Role of RAD52 Epistasis Group Genes in Homologous Recombination and Double-Strand Break Repair

  2002cited by this paper
• Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases.

  2002cited by this paper
• Targeted inactivation of p53 in human cells does not result in aneuploidy.

  2002cited by this paper
• Integration of splicing, transport and translation to achieve mRNA quality control by the nonsense-mediated decay pathway

  2002cited by this paper
• Rapid, high-throughput engineering of sequence-specific zinc finger DNA-binding proteins.

  2001cited by this paper
• A rapid, generally applicable method to engineer zinc fingers illustrated by targeting the HIV-1 promoter

  2001cited by this paper
• Regulation of an Endogenous Locus Using a Panel of Designed Zinc Finger Proteins Targeted to Accessible Chromatin Regions

  2001cited by this paper
• Expressing the human genome

  2001cited by this paper
• Stimulation of Homologous Recombination through Targeted Cleavage by Chimeric Nucleases

  2001cited by this paper
• Improved DNA binding specificity from polyzinc finger peptides by using strings of two-finger units.

  2001cited by this paper
• Design and selection of novel Cys2His2 zinc finger proteins.

  2001cited by this paper
• Advances in zinc finger engineering.

  2000cited by this paper
• Advances in Immunology: Primary Immunodeficiency Diseases Due to Defects in Lymphocytes

  2000cited by this paper
• Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

  2000cited by this paper
• Synthetic Zinc Finger Transcription Factor Action at an Endogenous Chromosomal Site

  2000cited by this paper
• Multiple Pathways of Recombination Induced by Double-Strand Breaks in Saccharomyces cerevisiae

  1999cited by this paper
• A detailed study of the substrate specificity of a chimeric restriction enzyme.

  1999cited by this paper
• Gene Conversion Tracts from Double-Strand Break Repair in Mammalian Cells

  1998cited by this paper
• Therapeutic gene targeting

  1998cited by this paper
• Bypass of senescence after disruption of p21CIP1/WAF1 gene in normal diploid human fibroblasts.

  1997cited by this paper
• Physical basis of a protein-DNA recognition code.

  1997cited by this paper
• Repair of site-specific double-strand breaks in a mammalian chromosome by homologous and illegitimate recombination

  1997cited by this paper
• Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain.

  1996cited by this paper
• Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae

  1995cited by this paper
• In vivo repression by a site-specific DNA-binding protein designed against an oncogenic sequence

  1994cited by this paper
• Expression of a site-specific endonuclease stimulates homologous recombination in mammalian cells.

  1994cited by this paper
• Co-chairman's remarks: protein designs for the specific recognition of DNA.

  1993cited by this paper
• Zinc finger-DNA recognition: crystal structure of a Zif268-DNA complex at 2.1 A

  1991cited by this paper
• High frequency targeting of genes to specific sites in the mammalian genome.

  1986cited by this paper
• Repetitive zinc‐binding domains in the protein transcription factor IIIA from Xenopus oocytes.

  1985cited by this paper

• Advances in Double‐Stranded DNA Targeting Technologies

  2026cites this paper
• Harnessing iPSCs, 3D organoids, and multiomics to model rare vascular diseases: Emerging new approach methodologies

  2026cites this paper
• [Advances in genome editing and lodging resistance in sugarcane].

  2026cites this paper
• Rational design of synthetic proteins using a genome-scale CRISPR screen

  2026cites this paper
• “Snip, snip, cure”? Philosophical, legal and biomedical perspectives on novel somatic genomic therapies

  2025cites this paper
• Retroviral Vector Technology for Gene Therapy: History, Current Landscape, and Future Prospects.

  2025cites this paper
• Mitochondrial Genetics in Cardiovascular Health and Disease: A Scientific Statement From the American Heart Association.

  2025cites this paper
• Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader

  2025cites this paper
• Gene Editing for Enhanced Swine Production: Current Advances and Prospects

  2025cites this paper
• CRISPR-based therapeutic genome editing for inherited blood disorders

  2025cites this paper
• CRISPR/Cas9: a sustainable technology to enhance climate resilience in major Staple Crops

  2025cites this paper
• Haplotype-resolved, gap-free genome assemblies provide insights into the divergence between Asian and European pears

  2025influential citation
• Photo‐Induced Ultra‐Fast Duplex Invasion Targeting Long‐Range Double‐Stranded DNA Using Artificial Nucleotide

  2025cites this paper
• Advances in gene therapy for mitochondrial genetic disorders: current status and clinical implementation challenges

  2025cites this paper
• Moving Therapeutic Genome Editing into Global Clinical Trials and Medicine

  2025cites this paper
• Mitochondrial diseases: from molecular mechanisms to therapeutic advances

  2025cites this paper
• Transgene integration in mammalian cells: The tools, the challenges, and the future.

  2025cites this paper
• Evaluation and prediction of guide RNA activities in genome-editing tools

  2025cites this paper
• Applications of multiplexed CRISPR–Cas for genome engineering

  2025cites this paper
• Recent Advances in Genome Editing and Bioinformatics: Addressing Challenges in Genome Editing Implementation and Genome Sequencing

  2025cites this paper
• Synergizing CRISPR-Cas9 with Advanced Artificial Intelligence and Machine Learning for Precision Drug Delivery: Technological Nexus and Regulatory Insights.

  2025cites this paper
• Mitochondrial Diseases: Molecular Pathogenesis and Therapeutic Advances

  2025cites this paper
• Rewiring tumor immunity via zinc finger proteins: a new frontier in cancer immunotherapy

  2025cites this paper
• The Strategy and Application of Gene Attenuation in Metabolic Engineering

  2025cites this paper
• Harnessing CRISPR/Cas9 system to engineer disease resistance in solanaceous crops: Current progress and future prospects

  2025cites this paper
• Gene transfer and genome editing of T cells for cancer immunotherapy: from allogeneic HSCT to TCR gene editing.

  2025cites this paper
• Precision scalpels for the epigenome: next-gen editing tools in targeted therapies

  2025cites this paper
• Modern approaches to the construction and use of recombinant viruses.

  2025cites this paper
• Risk Prediction of RNA Off-Targets of CRISPR Base Editors in Tissue-Specific Transcriptomes Using Language Models

  2025cites this paper
• Gene editing and reprogramming of human fibroblast cells (hFBs) to human pluripotent stem cells (hiPSCs).

  2024cites this paper
• Precise genome-editing in human diseases: mechanisms, strategies and applications

  2024cites this paper
• The impact of genomic distance on enhancer‐promoter interactions at the CFTR locus

  2024cites this paper
• Unexplored power of CRISPR-Cas9 in neuroscience, a multi-OMICs review.

  2024cites this paper
• Carmustine-Loaded Liposomal Delivery Effectively Targets Malignant Glioma Cells and Seizes Endothelial Sprouting In vitro

  2024cites this paper
• Progress Note 2024: Curing HIV; Not in My Lifetime or Just Around the Corner?

  2024cites this paper
• Knowledge domains and emerging trends of Genome-wide association studies in Alzheimer’s disease: A bibliometric analysis and visualization study from 2002 to 2022

  2024cites this paper
• CRISPR technologies for genome, epigenome and transcriptome editing

  2024influential citation
• Gene regulation in inborn errors of immunity: Implications for gene therapy design and efficacy

  2024cites this paper
• Small extracellular vesicles (sEVs)-based gene delivery platform for cell-specific CRISPR/Cas9 genome editing

  2024cites this paper
• Engineering Transcriptional Regulation for Cell-Based Therapies.

  2024cites this paper
• Genome Editing Approaches Using Zinc Finger Nucleases (ZFNs) for the Treatment of Motor Neuron Diseases.

  2024cites this paper
• A high efficiency precision genome editing method with CRISPR in iPSCs

  2024cites this paper
• CRISPR-based dissection of miRNA binding sites using isogenic cell lines is hampered by pervasive noise.

  2024cites this paper
• CRISPR/Cas9-Based Protocol for Precise Genome Editing in Induced Pluripotent Stem Cells

  2024cites this paper
• Characterization of Research Support of Genome Editing Technologies and Transition to Clinical Trials

  2024cites this paper
• Classical to modern biotechnology approaches, applications and future prospects in citrus breeding

  2024cites this paper
• CRISPR-based dissection of miRNA binding sites using isogenic cell lines is hampered by pervasive noise

  2024cites this paper
• Physiological basis for xenotransplantation from genetically modified pigs to humans

  2024cites this paper
• CRISPR/Cas9 Technology: A Novel Approach to Obesity Research.

  2024cites this paper
• Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1.

  2024cites this paper
• A structural peculiarity of Antarctic fish IgM drives the generation of an engineered mAb by CRISPR/Cas9

  2024cites this paper
• Advancements and challenges in developing in vivo CAR T cell therapies for cancer treatment

  2024cites this paper
• Genome Editing of Mammalian Cells Through RNA Transcript-Mediated Homologous Recombination Repair

  2024cites this paper
• A Story of Perseverance: An Interview with Matthew Porteus

  2024cites this paper
• Haplotype-resolved gapless genome and chromosome segment substitution lines facilitate gene identification in wild rice

  2024cites this paper
• Sleeping Beauty mRNA-LNP enables stable rAAV transgene expression in mouse and NHP hepatocytes and improves vector potency.

  2024cites this paper
• Erratum to: “Progress Note 2024: Curing HIV; Not in My Lifetime or Just Around the Corner? Pathogens and Immunity. 2024;8(2):115-157. doi: 10.20411/pai.v8i2.665”

  2024cites this paper
• Recent Therapeutic Gene Editing Applications to Genetic Disorders

  2024cites this paper
• Exploiting urine-derived induced pluripotent stem cells for advancing precision medicine in cell therapy, disease modeling, and drug testing

  2024cites this paper
• Gene Editing's Sharp Edge: Understanding Zinc Finger Nucleases (ZFN), Transcription Activator-Like Effector Nucleases (TALEN) and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)

  2024cites this paper
• A Story of Perseverance: An Interview with Matthew Porteus

  2024cites this paper
• Different Gene Therapies Fortreatment of Hemophilia A

  2023cites this paper
• Genetic Manipulation Approaches to Enhance the Clinical Application of NK Cell-Based Immunotherapy

  2023cites this paper
• CRISPR enables sustainable cereal production for a greener future.

  2023cites this paper
• Applications of Innovation Technologies for Personalized Cancer Medicine: Stem Cells and Gene-Editing Tools.

  2023cites this paper
• A new age of precision gene therapy.

  2023cites this paper
• Investigations on Antarctic fish IgM drives the generation of an engineered mAb by CRISPR/Cas9

  2023cites this paper
• Engineering CRISPR Cures: An Interview with Fyodor Urnov

  2023cites this paper
• Development of mitochondrial gene-editing strategies and their potential applications in mitochondrial hereditary diseases: a review.

  2023cites this paper
• Progressing nanotechnology to improve targeted cancer treatment: overcoming hurdles in its clinical implementation

  2023cites this paper
• Progress in gene editing tools, implications and success in plants: a review

  2023cites this paper
• The Regulation of Cell Therapy and Gene Therapy Products in Switzerland.

  2023cites this paper
• GENERATION OF A NUCLEASE TARGETING PARALLEL G-QUADRUPLEX FOR SPECIFIC CUTTING OF DOUBLE STRANDED DNA

  2023cites this paper
• Functional annotation of variants of the BRCA2 gene via locally haploid human pluripotent stem cells

  2023cites this paper
• In vitro evaluation of pH-sensitive liposomes loaded with d-limonene in targeting human malignant glioma cells

  2023cites this paper
• The emerging roles of long noncoding RNAs in lymphatic vascular development and disease

  2023cites this paper
• Using Ribonucleoprotein-based CRISPR/Cas9 to Edit Single Nucleotide on Human Induced Pluripotent Stem Cells to Model Type 3 Long QT Syndrome (SCN5A±)

  2023cites this paper
• A DddA ortholog-based and transactivator-assisted nuclear and mitochondrial cytosine base editors with expanded target compatibility.

  2023cites this paper
• High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis

  2023cites this paper
• Review: Recent Applications of Gene Editing in Fish Species and Aquatic Medicine

  2023cites this paper
• Nanobiomaterial vectors for improving gene editing and gene therapy

  2023cites this paper
• Correcting inborn errors of immunity: From viral mediated gene addition to gene editing

  2023cites this paper
• Zinc binding of a Cys2His2-type zinc finger protein is enhanced by the interaction with DNA

  2023cites this paper
• Plant breeding advancements with “CRISPR-Cas” genome editing technologies will assist future food security

  2023cites this paper
• Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood Disorders

  2023cites this paper
• Targeted DNA Demethylation: Vectors, Effectors and Perspectives

  2023cites this paper
• Nucleases in gene-editing technologies: past and prologue

  2023cites this paper
• An exosome-based gene delivery platform for cell-specific CRISPR/Cas9 genome editing

  2023cites this paper
• A Brief Review of Duchenne Muscular Dystrophy Treatment Options, with an Emphasis on Two Novel Strategies

  2023cites this paper
• Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.

  2023cites this paper
• Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells

  2023cites this paper
• The past, present, and future of artificial zinc finger proteins: design strategies and chemical and biological applications

  2023cites this paper
• Interactions of an Artificial Zinc Finger Protein with Cd(II) and Hg(II): Competition and Metal and DNA Binding

  2023cites this paper
• CRISPR Off-Target Analysis Platforms.

  2023cites this paper
• Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders

  2023cites this paper
• Rare immune diseases paving the road for genome editing-based precision medicine

  2023cites this paper
• Infertility control of transgenic fluorescent zebrafish with targeted mutagenesis of the dnd1 gene by CRISPR/Cas9 genome editing

  2023cites this paper
• Telomere-to-telomere pear (Pyrus pyrifolia) reference genome reveals segmental and whole genome duplication driving genome evolution

  2023influential citation
• Current Status and Future Strategies for Advancing Functional Circuit Mapping In Vivo

  2023cites this paper
• Virus-Like Particles Enable Low Levels of CRISPR-Cas9-Mediated Homology-Directed Repair

  2023cites this paper