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1. Full Text A New Class of Medicines through DNA Editing
by Porteus, Matthew H
The New England Journal of Medicine, ISSN 0028-4793, 03/2019, Volume 380, Issue 10, pp. 947 - 959
Therapeutic editing of the human genome has long been a holy grail of genetic medicine. Different approaches are founded on a bedrock of basic laboratory... 
MEDICINE, GENERAL & INTERNAL | MOUSE MODEL | DOUBLE-STRAND BREAKS | CRISPR-CAS9 | HOMOLOGOUS RECOMBINATION | TARGETED GENE ADDITION | ZINC-FINGER NUCLEASES | EXPRESSION | GENOMIC DNA | T-CELLS | HEMATOPOIETIC STEM | History, 21st Century | DNA | History, 20th Century | CRISPR-Cas Systems | Drug Discovery - methods | CRISPR-Associated Protein 9 | Gene Editing - methods | Gene Editing - history | Care and treatment | Genetic disorders | Research | T cells | CRISPR | Enzymes | Genetic counseling | Genes | Committees | Clinical trials | Genomes | Deoxyribonucleic acid--DNA | Immune system
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2. Genome editing
: the next step in gene therapy
by Cathomen, Toni, Herausgeber and Hirsch, Matthew, Herausgeber and Porteus, Matthew, Herausgeber
2016, 1st ed. 2016, Advances in experimental medicine and biology, ISBN 9781493935079, Volume 895, xvi, 263 pages
This comprehensive volume explores human genetic engineering its pre-clinical and clinical applications, current developments, and as treatment for hereditary... 
Gene therapy | Genetic engineering | Human genetics | Human Genetics | Biomedicine
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3. Genome Editing for the β-Hemoglobinopathies
by Porteus, Matthew H
Advances in Experimental Medicine and Biology, ISSN 0065-2598, 2017, Volume 1013, pp. 203 - 217
The β-hemoglobinopathies are diverse set of disorders caused by mutations in the β-globin (HBB) gene. Because HBB protein is a critical component (along with... 
Nucleases | Genome editing | Sickle cell disease | β-hemoglobinopathies | HBB gene | β-thalassemia
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4. Full Text Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factors
by Voit, Richard A and McMahon, Moira A and Sawyer, Sara L and Porteus, Matthew H
Molecular Therapy, ISSN 1525-0016, 2013, Volume 21, Issue 4, pp. 786 - 795
Restriction factors constitute a newly appreciated line of innate immune defense, blocking viral replication inside of infected cells. In contrast to these... 
MEDICINE, RESEARCH & EXPERIMENTAL | HUMAN STEM-CELLS | ACTIVATION | DEFECTS | PROTEIN | ZINC-FINGER NUCLEASES | DELIVERY | INDIVIDUALS | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | LENTIVIRAL VECTOR | GENETICS & HEREDITY | GENE-THERAPY | INFECTION | Cell Line | HIV Infections - immunology | Receptors, CCR5 - metabolism | T-Lymphocytes - metabolism | Humans | T-Lymphocytes - virology | CD4 Antigens - metabolism | Receptors, CXCR4 - metabolism
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5. Full Text AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs
by Dudek, Amanda M and Porteus, Matthew H
Molecular Therapy, ISSN 1525-0016, 10/2019, Volume 27, Issue 10, pp. 1701 - 1705
PROGENITOR CELLS | MEDICINE, RESEARCH & EXPERIMENTAL | GENE | RNA | INTEGRATION | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | ADENOASSOCIATED VIRUS VECTORS | PROMOTER | EXPRESSION | HEMATOPOIETIC STEM | NUCLEASES
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6. Full Text Towards a new era in medicine: Therapeutic genome editing
by Porteus, Matthew H
Genome Biology, ISSN 1474-7596, 12/2015, Volume 16, Issue 1, p. 286
Genome editing is the process of precisely modifying the nucleotide sequence of the genome. It has provided a powerful approach to research questions but, with... 
HEMATOPOIETIC STEM/PROGENITOR CELLS | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | EMBRYONIC STEM-CELLS | OFF-TARGET CLEAVAGE | IN-VIVO | GENETICS & HEREDITY | ADENOASSOCIATED VIRUS VECTORS | DOUBLE-STRAND BREAKS | DUCHENNE MUSCULAR-DYSTROPHY | HOMOLOGOUS RECOMBINATION | ZINC-FINGER NUCLEASES | MAMMALIAN-CELLS | Gene Targeting - methods | Animals | Genome | Humans | Genetic Therapy - methods | CRISPR | Genetic disorders | Infectious diseases | Yeast | Disease | Nucleotide sequence | Genes | Stem cells | Genomes | Genetic engineering | Mutation | Deoxyribonucleic acid--DNA | Review
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7. Full Text Plant biotechnology Zinc fingers on target
by Porteus, Matthew H
Nature, ISSN 0028-0836, 05/2009, Volume 459, Issue 7245, pp. 337 - 338
MAMMALIAN-CELLS | MULTIDISCIPLINARY SCIENCES | NUCLEASES | Gene Targeting - methods | Zinc Fingers | Plants, Genetically Modified - genetics | Humans | Mutagenesis, Site-Directed - methods | Breeding - methods | Deoxyribonucleases - metabolism | Biotechnology - methods | Animals | Genes, Plant - genetics | Recombination, Genetic - genetics | Food, Genetically Modified | Deoxyribonucleases - chemistry
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8. Full Text Gene targeting using zinc finger nucleases
by Porteus, Matthew H and Carroll, Dana
Nature Biotechnology, ISSN 1087-0156, 08/2005, Volume 23, Issue 8, pp. 967 - 973
The ability to achieve site-specific manipulation of the mammalian genome has widespread implications for basic and applied research. Gene targeting is a... 
SEVERE COMBINED IMMUNODEFICIENCY | CLEAVAGE DOMAIN | CHIMERIC RESTRICTION ENZYMES | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | EMBRYONIC STEM-CELLS | DNA-BINDING PROTEINS | DOUBLE-STRAND BREAKS | HOMING ENDONUCLEASES | HOMOLOGOUS RECOMBINATION | MAMMALIAN-CELLS | SACCHAROMYCES-CEREVISIAE | DNA - genetics | Genetic Engineering - methods | Deoxyribonucleases - genetics | Gene Targeting - methods | Zinc Fingers | DNA Damage - genetics | Recombination, Genetic - genetics | DNA - metabolism | DNA Repair - genetics | Deoxyribonucleases - metabolism
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9. Full Text 563. Analysis of DNA Repair Pathway Choice Upon Induction of Double-Strand Breaks by Engineered Nuclease
by Kumaran, R. Ileng and Li, Jingjing and Dynan, William S and Porteus, Matthew H and Spector, David L
Molecular Therapy, ISSN 1525-0016, 05/2016, Volume 24, pp. S225 - S225
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10. Full Text Chimeric Nucleases Stimulate Gene Targeting in Human Cells
by Matthew H. Porteus and David Baltimore
Science, ISSN 0036-8075, 5/2003, Volume 300, Issue 5620, pp. 763 - 763
Correction of gene defects in human somatic cells by targeting as has been used in murine embryonic stem cells has been precluded by the low spontaneous rate... 
Gene targeting | Somatic cells | DNA | Embryonic stem cells | Binding sites | Zinc | Brevia | RESTRICTION ENZYMES | HOMOLOGOUS RECOMBINATION | CLEAVAGE | MULTIDISCIPLINARY SCIENCES | Cell Line | Gene Targeting - methods | Green Fluorescent Proteins | Zinc Fingers | Humans | DNA - metabolism | Recombinant Fusion Proteins - chemistry | Nuclear Localization Signals | Deoxyribonucleases, Type II Site-Specific - genetics | Recombinant Fusion Proteins - metabolism | Deoxyribonucleases, Type II Site-Specific - metabolism | Deoxyribonucleases, Type II Site-Specific - chemistry | Transfection | Recombination, Genetic | Saccharomyces cerevisiae Proteins | Luminescent Proteins - genetics | Mutation | Dimerization | Genetic disorders | Research | Molecular genetics | Genes | Cells
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11. Full Text Mammalian gene targeting with designed zinc finger nucleases
by Porteus, Matthew H
Molecular Therapy, ISSN 1525-0016, 02/2006, Volume 13, Issue 2, pp. 438 - 446
Gene targeting by homologous recombination is a powerful method to manipulate the genome precisely and could be exploited to correct genetic defects. Zinc... 
gene targeting | gene therapy | zinc finger nucleases | homologous recombination | Zinc finger nucleases | Gene targeting | Gene therapy | Homologous recombination | MEDICINE, RESEARCH & EXPERIMENTAL | CHIMERIC RESTRICTION ENZYMES | SEQUENCES | DOUBLE-STRAND BREAKS | DNA-RECOGNITION | CLEAVAGE | INTRACHROMOSOMAL HOMOLOGOUS RECOMBINATION | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | EMBRYONIC STEM-CELLS | GENETICS & HEREDITY | PROTEINS | SELECTION | DOMAINS | Cell Line | Gene Targeting - methods | Mutagenesis, Insertional - methods | Humans | Gene Silencing | CD8 Antigens - biosynthesis | Green Fluorescent Proteins - genetics | Interleukin Receptor Common gamma Subunit - metabolism | Globins - metabolism | Interleukin Receptor Common gamma Subunit - genetics | Deoxyribonucleases - genetics | CD8 Antigens - metabolism | Globins - genetics | Deoxyribonucleases - biosynthesis | Green Fluorescent Proteins - biosynthesis | CD8 Antigens - genetics | Cloning, Molecular - methods | Plasmids - genetics | Deoxyribonucleases - physiology | Zinc Fingers - genetics | Plasmids - biosynthesis | Animal genetics | Genetic research | Nucleases | Zinc finger proteins | DNA binding proteins
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12. Full Text Erratum: Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6 (eLife (2017) 6 PII: e43690)
by Bak, Rasmus O and Dever, Daniel P and Reinisch, Andreas and Cruz Hernandez, David and Majeti, Ravindra and Porteus, Matthew H
eLife, 11/2018, Volume 7
stem cells | genomics | genetics | regenerative medicine
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13. Full Text Genome Engineering to Prospectively Investigate the Pathogenesis of MLL-AF9 Acute Leukemia
by Buechele, Corina and Breese, Erin H and Schneidawind, Dominik and Porteus, Matthew H and Cleary, Michael L
Blood, ISSN 0006-4971, 12/2014, Volume 124, Issue 21, pp. 883 - 883
Abstract Chromosomal rearrangements involving the MLL gene occur in both primary and treatment-related leukemias, and are associated with a poor prognosis.... 
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14. Full Text An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level
by Daniel E. Bauer and Sophia C. Kamran and Samuel Lessard and Jian Xu and Yuko Fujiwara and Carrie Lin and Zhen Shao and Matthew C. Canver and Elenoe C. Smith and Luca Pinello and Peter J. Sabo and Jeff Vierstra and Richard A. Voit and Guo-Cheng Yuan and Matthew H. Porteus and John A. Stamatoyannopoulos and Guillaume Lettre and Stuart H. Orkin
Science, ISSN 0036-8075, 10/2013, Volume 342, Issue 6155, pp. 253 - 257
Genome-wide association studies (GWASs) have ascertained numerous trait-associated common genetic variants, frequently localized to regulatory DNA. We found... 
Chromatin | Erythroblasts | Genetic variation | Transgenic animals | Lymphocytes | REPORTS | Cell lines | Alleles | Genomes | Erythroid cells | Gene expression | LYMPHOID DEVELOPMENT | DISSECTION | GLOBIN | DNA | MULTIDISCIPLINARY SCIENCES | DYNAMICS | SICKLE-CELL-DISEASE | HAPLOTYPE FUSION PCR | EXPRESSION | GENOME-WIDE ASSOCIATION | COMMON DISEASE | Chromatin - metabolism | Fetal Hemoglobin - biosynthesis | Humans | Precursor Cells, B-Lymphoid - metabolism | Genetic Variation | Chromatin Immunoprecipitation | Hemoglobinopathies - genetics | Nuclear Proteins - genetics | Gene Targeting | Genome-Wide Association Study | Cells, Cultured | Erythroid Cells - metabolism | Gene Expression Regulation | Chromosome Mapping | Transcription Factors - genetics | Hemoglobinopathies - therapy | Transcription Factors - metabolism | Carrier Proteins - genetics | Animals | Genetic Engineering | Enhancer Elements, Genetic | Fetal Hemoglobin - genetics | Cell Line, Tumor | Mice | Chromatin - genetics | Hemoglobin | Physiological aspects | Genetic aspects | Research | Nucleotide sequencing | DNA sequencing | Genetic diversity | Developmental biology | Fetuses | Erythrocytes
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15. Full Text Knock-in editing: It functionally corrects
by Porteus, Matthew H
Blood, ISSN 0006-4971, 05/2016, Volume 127, Issue 21, pp. 2507 - 2509
In this issue of Blood , Hubbard et al provide important proof-of-concept data on the utility of using genome editing to knock-in a wild-type (WT)... 
HEMATOLOGY | CELLS | GENOME | RNA Editing | Humans | Gene Editing | CRISPR-Cas Systems | Inside
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16. Full Text Activation of proto-oncogenes by disruption of chromosome neighborhoods
by Hnisz, Denes and Weintrau, Abraham S and Day, Daniel S and Valton, Anne-Laure and Bak, Rasmus O and Li, Charles H and Goldmann, Johanna and Lajoie, Bryan R and Fan, Zi Peng and Sigova, Alla A and Reddy, Jessica and Borges-Rivera, Diego and Lee, Tong Ihn and Jaenisch, Rudolf and Porteus, Matthew H and Dekker, Job and Young, Richard A
Science, ISSN 0036-8075, 03/2016, Volume 351, Issue 6280, pp. 1454 - 1458
Oncogenes are activated through well-known chromosomal alterations such as gene fusion, translocation, and focal amplification. In light of recent evidence... 
ONCOGENES | TOPOLOGY | 3D GENOME | SUPER-ENHANCERS | MULTIDISCIPLINARY SCIENCES | ACUTE LYMPHOBLASTIC-LEUKEMIA | CELL IDENTITY | TRANSCRIPTION | CANCER GENOME | ARCHITECTURE | REGULATORY LANDSCAPE | Sequence Deletion | Translocation, Genetic | Proto-Oncogenes - genetics | Precursor T-Cell Lymphoblastic Leukemia-Lymphoma - genetics | Humans | Transcriptional Activation | Chromosome Aberrations | HEK293 Cells | Chromosome Mapping | Gene Expression Regulation, Leukemic | Mutation | Genetic aspects | Research | Oncology, Experimental | Oncogenes | Cancer | Gene expression | Chromosomes | Genomics
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