Department of Pediatrics,
Center for Genomic Engineering,
Masonic Cancer Center,
Center for Genome Engineering,
Stem Cell Institute
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TALEN-based gene correction for epidermolysis bullosa.
Molecular therapy : the journal of the American Society of Gene Therapy Jun, 2013 | Pubmed ID: 23546300
DNA methylation of Runx1 regulatory regions correlates with transition from primitive to definitive hematopoietic potential in vitro and in vivo.
Blood Oct, 2013 | Pubmed ID: 24030384
Trp53 haploinsufficiency modifies EGFR-driven peripheral nerve sheath tumorigenesis.
The American journal of pathology Jul, 2014 | Pubmed ID: 24832557
Fanconi anemia gene editing by the CRISPR/Cas9 system.
Human gene therapy Feb, 2015 | Pubmed ID: 25545896
From marrow to matrix: novel gene and cell therapies for epidermolysis bullosa.
Molecular therapy : the journal of the American Society of Gene Therapy Jun, 2015 | Pubmed ID: 25803200
In Vitro T-Cell Generation From Adult, Embryonic, and Induced Pluripotent Stem Cells: Many Roads to One Destination.
Stem cells (Dayton, Ohio) Nov, 2015 | Pubmed ID: 26227158
Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases.
Molecular therapy : the journal of the American Society of Gene Therapy Mar, 2016 | Pubmed ID: 26502778
CRISPR/Cas9 Targeted Gene Editing and Cellular Engineering in Fanconi Anemia.
Stem cells and development 10, 2016 | Pubmed ID: 27538887
Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology.
Journal of inherited metabolic disease 03, 2017 | Pubmed ID: 27743312
CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa.
NPJ Regenerative medicine , 2016 | Pubmed ID: 28250968
Aryl hydrocarbon receptor inhibition promotes hematolymphoid development from human pluripotent stem cells.
Blood 06, 2017 | Pubmed ID: 28533309
CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells.
International journal of molecular sciences Jun, 2017 | Pubmed ID: 28613254
Dermatopontin in Bone Marrow Extracellular Matrix Regulates Adherence but Is Dispensable for Murine Hematopoietic Cell Maintenance.
Stem cell reports 09, 2017 | Pubmed ID: 28844660
Rapid generation of Col7a1 mouse model of recessive dystrophic epidermolysis bullosa and partial rescue via immunosuppressive dermal mesenchymal stem cells.
Laboratory investigation; a journal of technical methods and pathology 10, 2017 | Pubmed ID: 28892093
Rapid DNA replication origin licensing protects stem cell pluripotency.
eLife 11, 2017 | Pubmed ID: 29148972
CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression.
International journal of molecular sciences Mar, 2018 | Pubmed ID: 29565806
Engineering of Primary Human B cells with CRISPR/Cas9 Targeted Nuclease.
Scientific reports 08, 2018 | Pubmed ID: 30108345
Highly multiplexed genome engineering using CRISPR/Cas9 gRNA arrays.
PloS one , 2018 | Pubmed ID: 30222773
EditR: A Method to Quantify Base Editing from Sanger Sequencing.
The CRISPR journal 06, 2018 | Pubmed ID: 31021262
Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs.
The Journal of investigative dermatology 02, 2020 | Pubmed ID: 31437443
A Genetically Engineered Primary Human Natural Killer Cell Platform for Cancer Immunotherapy.
Molecular therapy : the journal of the American Society of Gene Therapy 01, 2020 | Pubmed ID: 31704085
Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors.
Nature communications 11, 2019 | Pubmed ID: 31745080
Author Correction: Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors.
Nature communications Dec, 2019 | Pubmed ID: 31811147
Efficient targeted integration directed by short homology in zebrafish and mammalian cells.
eLife May, 2020 | Pubmed ID: 32412410
CRISPR-Cas9 cytidine and adenosine base editing of splice-sites mediates highly-efficient disruption of proteins in primary and immortalized cells.
Nature communications Apr, 2021 | Pubmed ID: 33893286
Comparative international incidence of Ewing sarcoma 1988 to 2012.
International journal of cancer Sep, 2021 | Pubmed ID: 33961701
Engineering T cells to enhance 3D migration through structurally and mechanically complex tumor microenvironments.
Nature communications May, 2021 | Pubmed ID: 33990566
An irradiated marrow niche reveals a small noncollagenous protein mediator of homing, dermatopontin.
Blood advances Sep, 2021 | Pubmed ID: 34448828
MultiEditR: The first tool for the detection and quantification of RNA editing from Sanger sequencing demonstrates comparable fidelity to RNA-seq.
Molecular therapy. Nucleic acids Sep, 2021 | Pubmed ID: 34589274
A BAFF ligand-based CAR-T cell targeting three receptors and multiple B cell cancers.
Nature communications Jan, 2022 | Pubmed ID: 35017485
Myosin Heavy Chain Converter Domain Mutations Drive Early-Stage Changes in Extracellular Matrix Dynamics in Hypertrophic Cardiomyopathy.
Frontiers in cell and developmental biology , 2022 | Pubmed ID: 35784482
Correction of Fanconi Anemia Mutations Using Digital Genome Engineering.
International journal of molecular sciences Jul, 2022 | Pubmed ID: 35955545
Developing Bottom-Up Induced Pluripotent Stem Cell Derived Solid Tumor Models Using Precision Genome Editing Technologies.
The CRISPR journal Aug, 2022 | Pubmed ID: 35972367
Internal checkpoint regulates T cell neoantigen reactivity and susceptibility to PD1 blockade.
Med (New York, N.Y.) Oct, 2022 | Pubmed ID: 36007524
A Pan-RNase Inhibitor Enabling CRISPR-mRNA Platforms for Engineering of Primary Human Monocytes.
International journal of molecular sciences Aug, 2022 | Pubmed ID: 36077152
CRISPR-Cas9 base editors and their current role in human therapeutics.
Cytotherapy Mar, 2023 | Pubmed ID: 36635153
Germline T cell receptor exchange results in physiological T cell development and function.
Nature communications Feb, 2023 | Pubmed ID: 36726009
Ablation of SYK Kinase from Expanded Primary Human NK Cells via CRISPR/Cas9 Enhances Cytotoxicity and Cytokine Production.
Journal of immunology (Baltimore, Md. : 1950) Apr, 2023 | Pubmed ID: 36881874
Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II.
Molecular genetics and metabolism Apr, 2023 | Pubmed ID: 37023503
Cas9-induced targeted integration of large DNA payloads in primary human T cells via homology-mediated end-joining DNA repair.
Nature biomedical engineering Dec, 2024 | Pubmed ID: 38092857
Precision Enhancement of CAR-NK Cells through Non-Viral Engineering and Highly Multiplexed Base Editing.
bioRxiv : the preprint server for biology Mar, 2024 | Pubmed ID: 38496503
Evolution of the clinical-stage hyperactive TcBuster transposase as a platform for robust non-viral production of adoptive cellular therapies.
Molecular therapy : the journal of the American Society of Gene Therapy Jun, 2024 | Pubmed ID: 38627969
Development and testing of a versatile genome editing application reporter (V-GEAR) system.
Molecular therapy. Methods & clinical development Jun, 2024 | Pubmed ID: 38764780
Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1.
Molecular therapy : the journal of the American Society of Gene Therapy Jun, 2024 | Pubmed ID: 38781958
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders.
Molecular therapy : the journal of the American Society of Gene Therapy Nov, 2024 | Pubmed ID: 39367605
Efficient multiplex non-viral engineering and expansion of polyclonal γδ CAR-T cells for immunotherapy.
bioRxiv : the preprint server for biology Oct, 2024 | Pubmed ID: 39464114
Ablation of FAS confers allogeneic CD3 CAR T cells with resistance to rejection by T cells and natural killer cells.
Nature biomedical engineering Dec, 2024 | Pubmed ID: 39558141
CAR-T cell targeting three receptors on autoreactive B cells for systemic lupus erythematosus therapy.
Journal of autoimmunity Feb, 2025 | Pubmed ID: 39832454
Targeting the intracellular immune checkpoint CISH with CRISPR-Cas9-edited T cells in patients with metastatic colorectal cancer: a first-in-human, single-centre, phase 1 trial.
The Lancet. Oncology May, 2025 | Pubmed ID: 40315882
Precision enhancement of CAR-NK cells through non-viral engineering and highly multiplexed base editing.
Journal for immunotherapy of cancer May, 2025 | Pubmed ID: 40341025
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