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M. Porteus · SciRah

Researcher profile · Semantic Scholar

M. Porteus

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M. Porteus — researcher profile on SciRah

h-index: 64
Works: 248
Citations: 20K

Most-cited papers

Generation of human striatal organoids and cortico-striatal assembloids from human pluripotent stem cells
Yuki Miura, Min-Yin Li, Fikri Birey, K. Ikeda +7 more
2020Nature Biotechnology368 citations Open access
Semantic Scholar
CRISPR/Cas9 genome editing in human hematopoietic stem cells
R. Bak, D. Dever, M. Porteus
2018Nature Protocols287 citations Open access
Semantic Scholar
Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification
S. Vaidyanathan, Krist T. Azizian, A. K. M. Ashiqul Haque, Jordana M. Henderson +7 more
2018Molecular Therapy: Nucleic Acids222 citations Open access
Semantic Scholar
A New Class of Medicines through DNA Editing
M. Porteus
2019New England Journal of Medicine192 citations
Semantic Scholar
Neuronal Defects in a Human Cellular Model of 22q11.2 Deletion Syndrome
Themasap A. Khan, Omer Revah, A. Gordon, Se-Jin Yoon +23 more
2020Nature Medicine176 citations Open access
Semantic Scholar
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.
C. Antoniani, Vasco Meneghini, Annalisa Lattanzi, T. Felix +15 more
2018Blood176 citations Open access
Semantic Scholar
Loss of extreme long-range enhancers in human neural crest drives a craniofacial disorder
H. K. Long, M. Osterwalder, Ian C. Welsh, Karissa L. Hansen +18 more
2020Cell Stem Cell158 citations Open access
Semantic Scholar
SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.
É. Haddad, B. Logan, Linda M. Griffith, R. Buckley +61 more
2018Blood154 citations Open access
Semantic Scholar
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
Annalisa Lattanzi, J. Camarena, Premanjali Lahiri, H. Segal +19 more
2021Science Translational Medicine127 citations Open access
Semantic Scholar
High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia
S. Vaidyanathan, A. Salahudeen, Z. Sellers, Dawn T. Bravo +20 more
2019Cell Stem Cell122 citations Open access
Semantic Scholar
Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease
S. H. Park, Ciaran M. Lee, D. Dever, Timothy H. Davis +8 more
2019Nucleic Acids Research119 citations Open access
Semantic Scholar
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.
Annalisa Lattanzi, Vasco Meneghini, Giulia Pavani, F. Amor +11 more
2019Molecular Therapy111 citations Open access
Semantic Scholar
Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
A. Wilkinson, D. Dever, Ron Baik, J. Camarena +5 more
2020Nature Communications98 citations Open access
Semantic Scholar
High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition
S. Selvaraj, W. Feist, Sébastien Viel, S. Vaidyanathan +12 more
2023Nature Biotechnology78 citations Open access
Semantic Scholar
Genome editing of donor-derived T cells to generate allogeneic chimeric antigen receptor-modified T cells: optimizing αβ T-cell-depleted haploidentical hematopoietic stem cell transplantation
V. Wiebking, Ciaran M. Lee, Nathalie Mostrel, Premanjali Lahiri +5 more
2020Haematologica68 citations Open access
Semantic Scholar
Generating human artery and vein cells from pluripotent stem cells highlights the arterial tropism of Nipah and Hendra viruses
L. Ang, A. Nguyen, Kevin J Liu, Angela Chen +22 more
2022Cell67 citations Open access
Semantic Scholar
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells
M. Cromer, J. Camarena, Renata M. Martin, B. Lesch +19 more
2020Nature Medicine67 citations Open access
Semantic Scholar
Enhanced Homology-directed Repair for Highly Efficient Gene Editing in Hematopoietic Stem/Progenitor Cells.
S. D. de Ravin, J. Brault, R. Meis, Siyuan Liu +19 more
2021Blood62 citations Open access
Semantic Scholar
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus
S. Vaidyanathan, Ron Baik, Lu Chen, Dawn T. Bravo +16 more
2021bioRxiv62 citations Open access
Semantic Scholar
Cas9-AAV6-engineered human mesenchymal stromal cells improved cutaneous wound healing in diabetic mice
W. Srifa, Nina Kosaric, Alvaro Amorin, Othmane Jadi +5 more
2020Nature Communications62 citations Open access
Semantic Scholar
Human assembloids reveal the consequences of CACNA1G gene variants in the thalamocortical pathway
Ji-il Kim, Yuki Miura, Min-Yin Li, Omer Revah +8 more
202360 citations
Semantic Scholar
Metabolic engineering generates a transgene-free safety switch for cell therapy
V. Wiebking, J. O. Patterson, Renata M. Martin, Monica K. Chanda +4 more
2020Nature Biotechnology51 citations
Semantic Scholar
Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
Samantha G. Scharenberg, É. Poletto, K. Lucot, P. Colella +4 more
2020Nature Communications49 citations Open access
Semantic Scholar
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
Colin L. Sweeney, M. Pavel-Dinu, Uimook Choi, J. Brault +12 more
2021Gene Therapy46 citations Open access
Semantic Scholar
Functional significance of U2AF1 S34F mutations in lung adenocarcinomas
M. S. Esfahani, L. J. Lee, Young-Jun Jeon, Ryan A. Flynn +10 more
2019Nature Communications46 citations Open access
Semantic Scholar
Gene Editing Rescues in vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System
Cameron L. Gardner, M. Pavel-Dinu, K. Dobbs, M. Bosticardo +13 more
2021Journal of Clinical Immunology43 citations Open access
Semantic Scholar
Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing
J. Bonafont, Á. Mencía, E. Chacón-Solano, Wai Srifa +10 more
2021Molecular Therapy41 citations Open access
Semantic Scholar
Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells
M. Cromer, Valentin V. Barsan, Erich Jaeger, Mengchi Wang +8 more
2022Nature Communications39 citations Open access
Semantic Scholar
Combined lineage tracing and scRNA-seq reveals unexpected first heart field predominance of human iPSC differentiation
Francisco X. Galdos, Carissa Lee, Soah Lee, Sharon L. Paige +9 more
2021bioRxiv39 citations Open access
Semantic Scholar
MLL leukemia induction by t(9;11) chromosomal translocation in human hematopoietic stem cells using genome editing.
C. Schneidawind, Johan Jeong, D. Schneidawind, I. Kim +7 more
2018Blood Advances36 citations Open access
Semantic Scholar
Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34+ hematopoietic stem and progenitor cells.
M. Cromer, Kiran R. Majeti, G. Rettig, K. Murugan +6 more
2023Molecular Therapy35 citations Open access
Semantic Scholar
Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells
M. Pavel-Dinu, V. Wiebking, Beruh T. Dejene, W. Srifa +14 more
2019Nature Communications32 citations Open access
Semantic Scholar
CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells
D. Dever, Samantha G. Scharenberg, J. Camarena, Eric J. Kildebeck +12 more
2019iScience31 citations Open access
Semantic Scholar
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells
J. Brault, Taylor Liu, Siyuan Liu, Amanda Lawson +17 more
2023Frontiers in Immunology30 citations Open access
Semantic Scholar
The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells
Rajiv K. Sharma, D. Dever, Ciaran M. Lee, Armon Azizi +6 more
2021Nature Communications29 citations Open access
Semantic Scholar
High-efficiency CRISPR induction of t(9;11) chromosomal translocations and acute leukemias in human blood stem cells.
Johan Jeong, Astraea Jager, P. Domizi, M. Pavel-Dinu +8 more
2019Blood Advances29 citations Open access
Semantic Scholar
Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for Ex-Vivo Gene Editing
A. Dudek, M. Porteus
2021Frontiers in Immunology27 citations Open access
Semantic Scholar
Highly Efficient Editing of the Beta-Globin Gene in Patient Derived Hematopoietic Stem and Progenitor Cells to Treat Sickle Cell Disease
S. H. Park, Ciaran M. Lee, D. Dever, Timothy H. Davis +5 more
2018Blood25 citations Open access
Semantic Scholar
Investigation of Cas9 antibodies in the human eye
Marcus A. Toral, Carsten T. Charlesworth, Benjamin Ng, Teja Chemudupati +5 more
2022Nature Communications23 citations Open access
Semantic Scholar
Macrophage Subpopulation Dynamics Shift following Intravenous Infusion of Mesenchymal Stromal Cells.
Nina Kosaric, W. Srifa, C. Bonham, H. Kiwanuka +7 more
2020Molecular Therapy23 citations Open access
Semantic Scholar
Cedar Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe SCD
J. Kanter, J. Dipersio, P. Leavey, D. Shyr +7 more
2021Blood21 citations Open access
Semantic Scholar
Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development
P. Cannon, A. Asokan, A. Czechowicz, P. Hammond +9 more
2021Human Gene Therapy20 citations Open access
Semantic Scholar
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency
Sara Fañanas-Baquero, O. Quintana-Bustamante, D. Dever, O. Alberquilla +14 more
2021Molecular Therapy: Methods & Clinical Development17 citations Open access
Semantic Scholar
DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis
J. Adair, Mark R. Enstrom, Kevin G. Haworth, Lauren E. Schefter +6 more
2020Molecular Therapy: Methods & Clinical Development17 citations Open access
Semantic Scholar
Reengineering Ponatinib to Minimize Cardiovascular Toxicity
A. Hnatiuk, A. Bruyneel, Dhanir Tailor, Mallesh Pandrala +15 more
2022Cancer Research16 citations Open access
Semantic Scholar
Hematopoietic stem cell gene editing and expansion: state-of-the-art technologies and recent applications.
M. Haltalli, A. Wilkinson, Alejo E. Rodriguez-Fraticelli, M. Porteus
2021Experimental Hematology16 citations Open access
Semantic Scholar
Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells
M. Pavel-Dinu, V. Wiebking, Beruh T. Dejene, W. Srifa +14 more
2019Nature Communications15 citations Open access
Semantic Scholar
AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs.
A. Dudek, M. Porteus
2019Molecular Therapy13 citations Open access
Semantic Scholar
First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency
R. Agarwal, K. Weinberg, Hye-Sook Kwon, Ann Le +17 more
202011 citations Open access
Semantic Scholar
Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease
Li-Wen Xu, Premanjali Lahiri, Jason Skowronski, N. Bhatia +2 more
2023Molecular Therapy: Methods & Clinical Development10 citations Open access
Semantic Scholar

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