Affiliation: Stanford University
- Towards a new era in medicine: therapeutic genome editingMatthew H Porteus
Department of Pediatrics, Stanford University, Welch Road, Stanford, CA, 94305, USA
Genome Biol 16:286. 2015..Genome editing is being developed to treat not only monogenic diseases but also infectious diseases and diseases that have both a genetic and an environmental component. ..
- Genome Editing: A New Approach to Human TherapeuticsMatthew Porteus
Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University, Stanford, California 94305 email
Annu Rev Pharmacol Toxicol 56:163-90. 2016..Although such an approach could clearly prevent the birth of people with devastating and destructive genetic diseases, questions remain about whether human society is morally responsible enough to use this tool. ..
- Strategies to increase genome editing frequencies and to facilitate the identification of edited cellsMatthew Porteus
Hematology Oncology Stem Cell Transplantation Cancer Biology, Department of Pediatrics, School of Medicine, Stanford University, Lorry Lokey Stem Cell Research Building, G3045, MC 5462, 269 Campus Drive, Stanford, CA, 94305, USA
Methods Mol Biol 1239:281-9. 2015....
- Nuclease-mediated gene editing by homologous recombination of the human globin locusRichard A Voit
Department of Pediatrics, Stanford University, 1291 Welch Rd Stanford, CA 94305, USA and Department of Pediatrics, University of Texas Southwestern Medical Center, 5323 Harry Hines Blvd Dallas, TX 75390, USA
Nucleic Acids Res 42:1365-78. 2014....
- Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome ModificationKimberly A Wilson
1 Department of Biochemistry, University of Texas Southwestern Medical Center, Dallas, Texas, USA 2 Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA
Mol Ther Nucleic Acids 2:e88. 2013..These findings significantly expand the range of sites that can be potentially targeted by these custom-engineered proteins.Molecular Therapy - Nucleic Acids (2013) 2, e88; doi:10.1038/mtna.2013.13; published online 30 April 2013...
- Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT LocusKimberly A Wilson
1 Department of Biochemistry, University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, USA 2 Department of Pediatrics, University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, USA
Mol Ther Nucleic Acids 2:e87. 2013..Our work provides a foundation for using engineered ZF proteins (ZFPs) for modulation of the hTERT locus.Molecular Therapy - Nucleic Acids (2013) 2, e87; doi:10.1038/mtna.2013.12; published online 23 April 2013...
- 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 serotypeBrian L Ellis
Department of Biochemistry, University of Texas Southwestern Medical Center, Dallas, TX 75390 9148, USA
Virol J 10:74. 2013..Although transduction studies have been completed, the bulk of the studies have been done in vivo, and there has never been a comprehensive study of transduction ex vivo/in vitro...
- Homologous recombination-based gene therapy for the primary immunodeficienciesMatthew Porteus
Department of Pediatrics, Divisions of Cancer Biology, Hematology Oncology, Human Gene Therapy, Stanford University, Stanford, California, USA
Ann N Y Acad Sci 1246:131-40. 2011..In this review, I discuss the development of nuclease-stimulated, homologous recombination-based approaches as a novel gene therapy strategy for the primary immunodeficiencies...
- Design and testing of zinc finger nucleases for use in mammalian cellsMatthew Porteus
Department of Pediatrics and Biochemistry, UT Southwestern Medical Center, Dallas, TX, USA
Methods Mol Biol 435:47-61. 2008..This chapter describes how to identify potential targets for ZFN cutting, to make ZFNs to cut this target site, and how to test whether the newly designed ZFNs are active in a mammalian cell culture-based system...
- Gene therapy for primary immunodeficienciesEric Kildebeck
Department of Pediatrics, Stanford University School of Medicine, California 94305, USA
Curr Opin Pediatr 24:731-8. 2012..In this review, we discuss recent advances in gene therapy with an emphasis on strategies to improve safety, including the emergence of gene targeting technologies for the treatment of PIDs...