Porteus, Matthew H.

Identification of preexisting adaptive immunity to Cas9 proteins in humans

Charlesworth, C. T., Deshpande, P. S., Dever, D. P., Camarena, J., Lemgart, V. T., Cromer, M. K., Vakulskas, C. A., Collingwood, M. A., Zhang, L., Bode, N. M., et al. (2019) · Nature Medicine

CRISPR and Genetic EngineeringRNA regulation and disease

Cited by 1,093

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

Hendel, A., Bak, R. O., Clark, J. T., Kennedy, A. B., Ryan, D. E., Roy, S., Steinfeld, I., Lunstad, B. D., Kaiser, R. J., Wilkens, A. B., et al. (2015) · Nature Biotechnology

CRISPR and Genetic EngineeringRNA Interference and Gene Delivery

Cited by 1,090

Activation of proto-oncogenes by disruption of chromosome neighborhoods

Hnisz, D., Weintraub, A. S., Day, D. S., Valton, A.-L., Bak, R. O., Li, C. H., Goldmann, J., Lajoie, B. R., Fan, Z. P., Sigova, A. A., et al. (2016) · Science

Genomics and Chromatin DynamicsChromatin Remodeling and Cancer

Cited by 1,042

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

Dever, D. P., Bak, R. O., Reinisch, A., Camarena, J., Washington, G., Nicolas, C. E., Pavel-Dinu, M., Saxena, N., Wilkens, A. B., Mantri, S., et al. (2016) · Nature

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 864

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

Vakulskas, C. A., Dever, D. P., Rettig, G. R., Turk, R., Jacobi, A. M., Collingwood, M. A., Bode, N. M., McNeill, M. S., Yan, S., Camarena, J., et al. (2018) · Nature Medicine

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 782

Chimeric nucleases stimulate gene targeting in human cells

Porteus, M. H., & Baltimore, D. (2003) · Science

CRISPR and Genetic EngineeringDNA Repair Mechanisms

Cited by 741

An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level

Bauer, D. E., Kamran, S. C., Lessard, S., Xu, J., Fujiwara, Y., Lin, C., Shao, Z., Canver, M. C., Smith, E. C., Pinello, L., et al. (2013) · Science

Hemoglobinopathies and Related DisordersGenomics and Chromatin Dynamics

Cited by 653

Gene targeting using zinc finger nucleases

Porteus, M. H., & Carroll, D. (2005) · Nature Biotechnology

CRISPR and Genetic EngineeringRNA Interference and Gene Delivery

Cited by 641

CRISPR/Cas9 genome editing in human hematopoietic stem cells.

Bak, R. O., Dever, D. P., & Porteus, M. H. (2018) · Nature Protocols

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 319

Mutations in the nuclear bile acid receptor FXR cause progressive familial intrahepatic cholestasis

Gomez-Ospina, N., Potter, C. J., Xiao, R., Manickam, K., Kim, M.-S., Kim, K. H., Shneider, B. L., Picarsic, J. L., Jacobson, T. A., Zhang, J., et al. (2016) · Nature Communications

Drug Transport and Resistance MechanismsPediatric Hepatobiliary Diseases and Treatments

Cited by 300

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification

Vaidyanathan, S., Azizian, K. T., Haque, A. K. M. A., Henderson, J. M., Hendel, A., Shore, S., Antony, J. S., Hogrefe, R. I., Kormann, M. S. D., Porteus, M. H., et al. (2018) · Molecular Therapy Nucleic Acids

CRISPR and Genetic EngineeringRNA Interference and Gene Delivery

Cited by 262

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype

Ellis, B. L., Hirsch, M. L., Barker, J. C., Connelly, J. P., Steininger, R. J., & Porteus, M. H. (2013) · Virology Journal

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 252

A New Class of Medicines through DNA Editing

Porteus, M. H. (2019) · New England Journal of Medicine

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 229

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Antoniani, C., Meneghini, V., Lattanzi, A., Felix, T., Romano, O., Magrin, E., Weber, L., Pavani, G., El Hoss, S., Kurita, R., et al. (2018) · Blood

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 194

Neuronal defects in a human cellular model of 22q11.2 deletion syndrome

Khan, T. A., Revah, O., Gordon, A., Yoon, S.-J., Krawisz, A. K., Goold, C., Sun, Y., Kim, C. H., Tian, Y., Li, M.-Y., et al. (2020) · Nature Medicine

Pluripotent Stem Cells ResearchGenomic variations and chromosomal abnormalities

Cited by 186

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

Bak, R. O., Dever, D. P., Reinisch, A., Cruz Hernandez, D., Majeti, R., & Porteus, M. H. (2017) · eLife

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 122

Generation of an HIV resistant T-cell line by targeted “stacking” of restriction factors

Voit, R. A., McMahon, M. A., Sawyer, S. L., & Porteus, M. H. (2013) · Molecular Therapy

HIV Research and TreatmentCRISPR and Genetic Engineering

Cited by 74

Towards a new era in medicine: therapeutic genome editing

Porteus, M. H. (2015) · Genome Biology

CRISPR and Genetic EngineeringRNA regulation and disease

Cited by 69

The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation

Dever, D. P., & Porteus, M. H. (2017) · Current Opinion in Hematology

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 62

MLL leukemia induction by genome editing of human CD34+ hematopoietic cells

Buechele, C., Breese, E. H., Schneidawind, D., Lin, C.-H., Jeong, J., Duque-Afonso, J., Wong, S. H. K., Smith, K. S., Negrin, R. S., Porteus, M., et al. (2015) · Blood

Acute Myeloid Leukemia ResearchAcute Lymphoblastic Leukemia research

Cited by 60

Human assembloids reveal the consequences of CACNA1G gene variants in the thalamocortical pathway

Kim, J.-I., Miura, Y., Li, M.-Y., Revah, O., Selvaraj, S., Birey, F., Meng, X., Thete, M. V., Pavlov, S. D., Andersen, J., et al. (2024) · Neuron

Ion channel regulation and functionNeural dynamics and brain function

Cited by 59

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

Hirsch, M. L., Fagan, B. M., Dumitru, R., Bower, J. J., Yadav, S., Porteus, M. H., Pevny, L. H., & Samulski, R. J. (2011) · PLoS ONE

Virus-based gene therapy researchDNA Repair Mechanisms

Cited by 53

Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34(+) hematopoietic stem and progenitor cells

Cromer, M. K., Majeti, K. R., Rettig, G. R., Murugan, K., Kurgan, G. L., Bode, N. M., Hampton, J. P., Vakulskas, C. A., Behlke, M. A., & Porteus, M. H. (2023) · Molecular Therapy

CRISPR and Genetic EngineeringRNA regulation and disease

Cited by 42

Investigation of Cas9 Antibodies in the Human Eye

Toral, M. A., Charlesworth, C. T., Ng, B., Chemudupati, T., Homma, S., Nakauchi, H., Bassuk, A. G., Porteus, M. H., & Mahajan, V. B. (2022) · Nature Communications

CRISPR and Genetic EngineeringOcular Diseases and Behçet’s Syndrome

Cited by 28