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Author Details

Matthew H Porteus
Stanford University
1984
173
61
Prashant Mali (CM4AI)
PMIDPaper TitleJournal TitlePublished Year
37537500High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition.Nat Biotechnol2024
38019608Small-molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states.Mol Biol Cell2024
36793210Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34<sup>+</sup> hematopoietic stem and progenitor cells.Mol Ther2023
37745470Small molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states.bioRxiv2023
37773046Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy.J Exp Med2023
38091985iPSC-derived hypoimmunogenic tissue resident memory T cells mediate robust anti-tumor activity against cervical cancer.Cell Rep Med2023
37637384Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease.Mol Ther Methods Clin Dev2023
36685559CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.Front Immunol2023
34735685Treatment decision-making in sickle cell disease patients.J Community Genet2022
35773054A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System.Hematol Oncol Clin North Am2022
35738284Generating human artery and vein cells from pluripotent stem cells highlights the arterial tropism of Nipah and Hendra viruses.Cell2022
35763671Reengineering Ponatinib to Minimize Cardiovascular Toxicity.Cancer Res2022
35414059Author Correction: Investigation of Cas9 antibodies in the human eye.Nat Commun2022
35953477Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells.Nat Commun2022
33794364Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.Mol Ther2022
35227602Design of experiments as a decision tool for cell therapy manufacturing.Cytotherapy2022
35217666Investigation of Cas9 antibodies in the human eye.Nat Commun2022
34973360Hematopoietic stem cell gene editing and expansion: State-of-the-art technologies and recent applications.Exp Hematol2022
32241852Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αβ T cell-depleted haploidentical hematopoietic stem cell transplantation.Haematologica2021
33623984Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells.Blood2021
33737751Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.Nat Med2021
33712802Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.Gene Ther2021
33678249Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex.Mol Ther2021
33650026Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System.J Clin Immunol2021
33514718Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice.Nat Commun2021
33821398Correction to: Gene Editing Rescues in Vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System.J Clin Immunol2021
33609734Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.Mol Ther2021
34485608Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.Mol Ther Methods Clin Dev2021
34122418Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for <i>Ex-Vivo</i> Gene Editing.Front Immunol2021
34232639An Unusual "OR" Gate for Allosteric Regulation of Mammalian Transglutaminase 2 in the Extracellular Matrix.J Am Chem Soc2021
34135108Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.Sci Transl Med2021
33427035Safe and Effective <i>In Vivo</i> Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development.Hum Gene Ther2021
33075720The Binns Program for Cord Blood Research: A novel model of cord blood banking for academic biomedical research.Placenta2021
33473139The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells.Nat Commun2021
31513279Early Tacrolimus Concentrations After Lung Transplant Are Predicted by Combined Clinical and Genetic Factors and Associated With Acute Kidney Injury.Clin Pharmacol Ther2020
31839569High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia.Cell Stem Cell2020
33136125CD34 expression does not correlate with immunophenotypic stem cell or progenitor content in human cord blood products.Blood Adv2020
33273741Generation of human striatal organoids and cortico-striatal assembloids from human pluripotent stem cells.Nat Biotechnol2020
32661439Metabolic engineering generates a transgene-free safety switch for cell therapy.Nat Biotechnol2020
32991838Loss of Extreme Long-Range Enhancers in Human Neural Crest Drives a Craniofacial Disorder.Cell Stem Cell2020
32820153Author Correction: Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.Nat Commun2020
32620863Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.Nat Commun2020
32989314Neuronal defects in a human cellular model of 22q11.2 deletion syndrome.Nat Med2020
32355868DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis.Mol Ther Methods Clin Dev2020
32494707CRISPR-based gene editing enables <i>FOXP3</i> gene repair in IPEX patient cells.Sci Adv2020
32483127Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards.Nat Commun2020
32424320Cas9-AAV6-engineered human mesenchymal stromal cells improved cutaneous wound healing in diabetic mice.Nat Commun2020
32531238Macrophage Subpopulation Dynamics Shift following Intravenous Infusion of Mesenchymal Stromal Cells.Mol Ther2020
30692695Identification of preexisting adaptive immunity to Cas9 proteins in humans.Nat Med2019
31836708Functional significance of U2AF1 S34F mutations in lung adenocarcinomas.Nat Commun2019
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Collaborators

John L R Rubenstein
UCSF Medical School
Co-authored papers 10
Shondra M Pruett-Miller
St. Jude Children's Research Hospital.
Co-authored papers 8
Hiromitsu Nakauchi
Advanced Research Institute, Tokyo Medical and Dental University
Co-authored papers 7
Ravindra Majeti
Stanford University School of Medicine
Co-authored papers 7
J Keith Joung
Massachusetts General Hospital
Co-authored papers 6
Vittorio Sebastiano
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Co-authored papers 4
Stacey Thibodeau-Beganny
Amherst College
Co-authored papers 3
Xiaolin Wu
Leidos Biomedical Research Inc.
Co-authored papers 3
Morgan L Maeder
Massachusetts General Hospital, Harvard Medical School
Co-authored papers 3
Nivedita Saxena
Co-authored papers 3
Sandra Burkett
Center for Cancer Research, National Cancer Institute, National Institutes of Health
Co-authored papers 2
Vivien A Sheehan
Co-authored papers 2
Jayakar V Nayak
Co-authored papers 2
Linzhao Cheng
Johns Hopkins University School of Medicine
Co-authored papers 2
Thomas J Montine
Stanford University
Co-authored papers 2
James A Olzmann
Co-authored papers 2
Benjamin P Kleinstiver
Center for Genomic Medicine, Massachusetts General Hospital
Co-authored papers 2
Hanns Lochm??ller
Children's Hospital of Eastern Ontario Research Institute
Co-authored papers 1
John A Stamatoyannopoulos
Altius Institute for Biomedical Sciences
Co-authored papers 1
Diego Borges-Rivera
Whitehead Institute for Biomedical Research, Massachusetts Institute of Technology
Co-authored papers 1
Larysa H Pevny
Co-authored papers 1
Ravinder J Singh
Co-authored papers 1
Matthew D Weitzman
The Children's Hospital of Philadelphia
Co-authored papers 1
William L Stanford
Ottawa Hospital Research Institute
Co-authored papers 1
Benhur Lee
Icahn School of Medicine at Mount Sinai
Co-authored papers 1
Zara M Patel
Co-authored papers 1
Pengfei Liu
Co-authored papers 1
Yaping Yang
Baylor College of Medicine
Co-authored papers 1
Aaron M Newman
Stanford University
Co-authored papers 1
Jeremy M Rock
The Rockefeller University
Co-authored papers 1
Release 1.2