Matthew H. Porteus

Also affiliated with California Institute for Regenerative Medicine, Institute for Stem Cell Biology and Regenerative Medicine, Stanford Medicine, Stanford University

Biochemistry, Genetics and Molecular BiologyMedicine

Pilot corpus only

This score is computed over theSindex pilot corpus and does not cover the full scientific literature. Scores are relative to papers we have ingested — papers, authors, and institutions outside the pilot are not represented. Methodology.

Top 100%percentile

0Author DataRank

Indexed papers

0in pilot corpus

datarank_citation_only_1hop_v6· scope data_onlyMethodology

Papers

Driven by 45 papers. Top paper: “Lineage-tracing hematopoietic stem cell origins in vivo to efficiently make human HLF+ HOXA+ hematopoietic progenitors from pluripotent stem cells”.

Lineage-tracing hematopoietic stem cell origins in vivo to efficiently make human HLF+ HOXA+ hematopoietic progenitors from pluripotent stem cells

Developmental Cell(2024)10.1016/j.devcel.2024.03.003

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0.894DataRank · unranked

46 citations

Sherry Li Zheng, Alana Nguyen, Angela Chen, Xiaochen Xiong, Timothy Chai +21 more

Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial

Nature Medicine(2025)10.1038/s41591-025-03817-1

N/A

0.432DataRank · unranked

15 citations

Alice Bertaina, Charmaine Soco, Janel Long-Boyle, Gopin Saini, Nivedita Kunte +26 more

Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells

Cell Reports(2025)10.1016/j.celrep.2024.115141

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0.399DataRank · unranked

11 citations

Eric Soupène, Devesh Sharma, Roshani Sinha, Travis McCreary, Britney Hernandez +20 more

DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis

Molecular Therapy — Methods & Clinical Development(2020)10.1016/j.omtm.2020.03.021

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0DataRank · unranked

19 citations

Mark Enstrom, Kevin G. Haworth, Lauren E. Schefter, Reza Shahbazi, Daniel Humphrys +5 more

Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase

Nature Communications(2020)10.1038/s41467-020-17100-z

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0DataRank · unranked

18 citations

Kavitha Siva, J. Chen, Gianluca Varetti, M. Youn, Hee‐Don Chae +16 more

Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex

Molecular Therapy(2020)10.1016/j.ymthe.2020.10.020

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0DataRank · unranked

13 citations

Francesco Puzzo, Mara Pavel-Dinu, Feijie Zhang, Sirika Pillay, Karim Majzoub +10 more

Macrophage Subpopulation Dynamics Shift following Intravenous Infusion of Mesenchymal Stromal Cells

Molecular Therapy(2020)10.1016/j.ymthe.2020.05.022

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0DataRank · unranked

27 citations

Waracharee Srifa, Clark A. Bonham, Harriet Kiwanuka, Kellen Chen, Britta Kuehlmann +6 more

Cas9-AAV6 Gene Correction of Beta-Globin in Autologous HSCs Improves Sickle Cell Disease Erythropoiesis in Mice

bioRxiv (Cold Spring Harbor Laboratory)(2020)10.1101/2020.10.13.338319

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0DataRank · unranked

11 citations

Daniel P. Dever, Ron Baik, Joab Camarena, Ian Hsu, Carsten T. Charlesworth +4 more

TRACE-Seq Reveals Clonal Reconstitution Dynamics of Gene Targeted Human Hematopoietic Stem Cells

bioRxiv (Cold Spring Harbor Laboratory)(2020)10.1101/2020.05.25.115329

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0DataRank · unranked

3 citations

Daniel P. Dever, Ciaran M. Lee, Armon Azizi, Yidan Pan, Joab Camarena +5 more

Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus

UNC Libraries(2020)10.17615/g4py-ja27

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0DataRank · unranked

0 citations

Kimberly Wilson, Matthew H. Porteus, Morgan Chateau

Viral Single-Strand DNA Induces p53-Dependent Apoptosis in Human Embryonic Stem Cells

UNC Libraries(2020)10.17615/5zwr-a987

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0DataRank · unranked

0 citations

Swati Yadav, Matthew L. Hirsch, Larysa Pevny, Jacquelyn J. Bower, Matthew H. Porteus +3 more

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Science Translational Medicine(2021)10.1126/scitranslmed.abf2444

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0DataRank · unranked

163 citations

Joab Camarena, Premanjali Lahiri, Helen Segal, Waracharee Srifa, Christopher A. Vakulskas +18 more

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

Nature Communications(2021)10.1038/s41467-021-20909-x

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0DataRank · unranked

101 citations

Daniel P. Dever, Ron Baik, Joab Camarena, Ian Hsu, Carsten T. Charlesworth +4 more

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

Nature Medicine(2021)10.1038/s41591-021-01284-y

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0DataRank · unranked

84 citations

Joab Camarena, Renata M. Martin, Benjamin J. Lesch, Christopher A. Vakulskas, Nicole M. Bode +18 more

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Blood(2021)10.1182/blood.2020008503

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0DataRank · unranked

74 citations

Julie Brault, Ronald J. Meis, Siyuan Liu, Linhong Li, Mara Pavel-Dinu +18 more

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus

Molecular Therapy(2021)10.1016/j.ymthe.2021.03.023

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0DataRank · unranked

69 citations

Ron Baik, Lü Chen, Dawn T. Bravo, Carlos J. Suarez, Shayda M. Abazari +15 more

Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System

Journal of Clinical Immunology(2021)10.1007/s10875-021-00989-6

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0DataRank · unranked

42 citations

Mara Pavel-Dinu, Kerry Dobbs, Marita Bosticardo, Paul K. Reardon, Justin Lack +12 more

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells

Nature Communications(2021)10.1038/s41467-020-20792-y

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0DataRank · unranked

30 citations

Daniel P. Dever, Ciaran M. Lee, Armon Azizi, Yidan Pan, Joab Camarena +5 more

Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for Ex-Vivo Gene Editing

Frontiers in Immunology(2021)10.3389/fimmu.2021.660302

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0DataRank · unranked

29 citations

Matthew H. Porteus, Amanda M. Dudek

Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair

Gene Therapy(2021)10.1038/s41434-021-00251-z

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0DataRank · unranked

52 citations

Mara Pavel-Dinu, Uimook Choi, Julie Brault, Taylor Liu, Sherry Koontz +11 more

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus

bioRxiv (Cold Spring Harbor Laboratory)(2021)10.1101/2021.02.26.432961

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0DataRank · unranked

8 citations

Ron Baik, Lü Chen, Dawn T. Bravo, Carlos J. Suarez, Shayda M. Abazari +15 more

Combined Lineage Tracing and scRNA-seq Reveals Unexpected First Heart Field Predominance of Human iPSC Differentiation

bioRxiv (Cold Spring Harbor Laboratory)(2021)10.1101/2021.09.30.462465

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0DataRank · unranked

3 citations

Carissa Lee, Soah Lee, William R. Goodyer, Sharon L. Paige, Gabriela V Escobar +7 more

Generating human artery and vein cells from pluripotent stem cells highlights the arterial tropism of Nipah and Hendra viruses

Cell(2022)10.1016/j.cell.2022.05.024

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0DataRank · unranked

87 citations

Alana Nguyen, Kevin J. Liu, Angela Chen, Xiaochen Xiong, Matthew B. Curtis +21 more

Investigation of Cas9 antibodies in the human eye

Nature Communications(2022)10.1038/s41467-022-28674-1

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0DataRank · unranked

27 citations

Carsten T. Charlesworth, Benjamin Ng, Teja Chemudupati, Shota Homma, Hiromitsu Nakauchi +4 more

Reengineering Ponatinib to Minimize Cardiovascular Toxicity

Cancer Research(2022)10.1158/0008-5472.can-21-3652

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0DataRank · unranked

19 citations

Arne A.N. Bruyneel, Dhanir Tailor, Mallesh Pandrala, Arpit Dheeraj, Wenqi Li +14 more

Genetically Corrected RAG2 -SCID Human Hematopoietic Stem Cells Restore V(D)J-Recombinase and Rescue Lymphoid Deficiency

bioRxiv (Cold Spring Harbor Laboratory)(2022)10.1101/2022.07.12.499831

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0DataRank · unranked

3 citations

Cameron L. Gardner, Yusuke Nakauchi, Tomoki Kawai, Ottavia M. Delmonte, Boaz Palterer +16 more

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells

Frontiers in Immunology(2023)10.3389/fimmu.2022.1067417

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0DataRank · unranked

26 citations

Taylor Liu, Siyuan Liu, Amanda Lawson, Uimook Choi, Nikita Kozhushko +16 more

Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease

Molecular Therapy — Methods & Clinical Development(2023)10.1016/j.omtm.2023.07.009

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0DataRank · unranked

12 citations

Premanjali Lahiri, Jason Skowronski, Neehar Bhatia, Annalisa Lattanzi, Matthew H. Porteus +1 more

Small-molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states

Molecular Biology of the Cell(2023)10.1091/mbc.e23-08-0336

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0DataRank · unranked

9 citations

Magda Wąchalska, Kirandeep K. Deol, Anais K. Amaya, Matthew H. Porteus, James A. Olzmann +2 more

Genetically corrected RAG2 -SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

Blood Advances(2023)10.1182/bloodadvances.2023011766

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0DataRank · unranked

13 citations

Cameron L. Gardner, Yusuke Nakauchi, Tomoki Kawai, Ottavia M. Delmonte, Boaz Palterer +18 more

Dual α-globin and truncated EPO receptor knockin restores hemoglobin production in α-thalassemia-derived red blood cells

bioRxiv (Cold Spring Harbor Laboratory)(2023)10.1101/2023.09.01.555926

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0DataRank · unranked

3 citations

Eric Soupène, Beeke Wienert, Han Yin, Devesh Sharma, Travis McCreary +9 more

Transient Inhibition of 53BP1 Increases the Frequency of Targeted Integration in Human Hematopoietic Stem and Progenitor Cells

Research Square(2023)10.21203/rs.3.rs-2621625/v1

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0DataRank · unranked

2 citations

M. Kyle Cromer, Steve E. Glenn, Christopher A. Vakulskas, Amanda M. Dudek, William N. Feist +4 more

Small molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states

bioRxiv (Cold Spring Harbor Laboratory)(2023)10.1101/2023.09.15.556420

N/A

0DataRank · unranked

0 citations

Magda Wąchalska, Kirandeep K. Deol, Anais K. Amaya, Matthew H. Porteus, James A. Olzmann +2 more

Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells

Nature Communications(2024)10.1038/s41467-023-43413-w

N/A

0DataRank · unranked

27 citations

M. Kyle Cromer, Steve E. Glenn, Christopher A. Vakulskas, Kay O. Chmielewski, Amanda M. Dudek +7 more

A simultaneous knockout knockin genome editing strategy in HSPCs potently inhibits CCR5- and CXCR4-tropic HIV-1 infection

Cell stem cell(2024)10.1016/j.stem.2024.03.002

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0DataRank · unranked

15 citations

William N. Feist, Elena J. Sasu, Sofia E. Luna, Kaya Ben-Efraim, Rasmus O. Bak +3 more

Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV

Molecular Therapy — Nucleic Acids(2024)10.1016/j.omtn.2024.102134

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0DataRank · unranked

13 citations

Jenny L. Kerschner, Alekh Paranjapye, Vrishti Sinha, Brian Lin, Tracy A. Bedrosian +5 more

Failure of metabolic checkpoint control during late-stage granulopoiesis drives neutropenia in reticular dysgenesis

Blood(2024)10.1182/blood.2024024123

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0DataRank · unranked

3 citations

Martin Arreola, Thomas P. Mathews, Andrew DeVilbiss, Zhiyu Zhao, Misty S. Martin-Sandoval +13 more

Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic Antibodies

bioRxiv (Cold Spring Harbor Laboratory)(2024)10.1101/2024.03.08.583956

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0DataRank · unranked

1 citations

Sofia E. Luna, Kaya Ben-Efraim, Maria V. Filsinger Interrante, Nelson A. Amorin, Nicole M. Johnston +6 more

Engineering inducible signaling receptors to enable erythropoietin-free erythropoiesis

bioRxiv (Cold Spring Harbor Laboratory)(2024)10.1101/2024.04.12.589260

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0DataRank · unranked

0 citations

Kiran R. Majeti, Freja K. Ekman, Sridhar Selvaraj, Eric Soupène, Prathamesh Chati +11 more

Engineering inducible signaling receptors to enable erythropoietin-free erythropoiesis

Research Square(2024)10.21203/rs.3.rs-4259044/v1

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0DataRank · unranked

0 citations

Aadit P. Shah, Kiran R. Majeti, Freja K. Ekman, Sridhar Selvaraj, Eric Soupène +10 more

Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis

Research Square(2024)10.21203/rs.3.rs-4986623/v1

N/A

0DataRank · unranked

0 citations

Aadit P. Shah, Kiran R. Majeti, Freja K. Ekman, Sridhar Selvaraj, Devesh Sharma +11 more

Loss of Extreme Long-Range Enhancers in Human Neural Crest Drives a Craniofacial Disorder

Cell stem cell(2020)10.1016/j.stem.2020.09.001

N/A

0DataRank · unranked

179 citations

Marco Osterwalder, Ian Welsh, Karissa Hansen, James Davies, Yiran E. Liu +17 more

Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis

Nature Communications(2025)10.1038/s41467-025-56239-5

N/A

0DataRank · unranked

7 citations

Kiran R. Majeti, Freja K. Ekman, Sridhar Selvaraj, Devesh Sharma, Roshani Sinha +11 more

CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

Science Advances(2020)10.1126/sciadv.aaz0571

N/A

0DataRank · unranked

114 citations

Ciaran M. Lee, Uma Lakshmanan, Suzette Shipp, Luise Froessl, Federica Barzaghi +16 more

Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards

Nature Communications(2020)10.1038/s41467-020-16455-7

N/A

0DataRank · unranked

113 citations

Jonas L. Fowler, M. Kyle Cromer, Benjamin J. Lesch, Ezequiel Ponce, Nobuko Uchida +4 more