Mark A Kay

Mark A Kay

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Mark A Kay

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Allele-Specific Silencing Ameliorates Restrictive Cardiomyopathy Attributable to a Human Myosin Regulatory Light Chain Mutation.

Circulation 2019 Aug 18;140(9):765-778. Epub 2019 Jul 18.

Division of Cardiovascular Medicine (K.Z.-R., A.D., P.C., G.R., C.S., J.L., T.F., W.N.P., S.S., K.S., N.S., N.J., Y.H., M.T.W., E.A.A.), Stanford University School of Medicine, CA.

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http://dx.doi.org/10.1161/CIRCULATIONAHA.118.036965DOI Listing
August 2019

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.

Mol Ther 2017 11 25;25(11):2477-2489. Epub 2017 Sep 25.

Department of Pediatrics and Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2017.09.020DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5675605PMC
November 2017

Sequence-Modified Antibiotic Resistance Genes Provide Sustained Plasmid-Mediated Transgene Expression in Mammals.

Mol Ther 2017 05 30;25(5):1187-1198. Epub 2017 Mar 30.

Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA 94305, USA; Department of Genetics, Stanford University School of Medicine, Palo Alto, CA 94305, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2017.03.003DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5417794PMC
May 2017

Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond.

Hum Gene Ther 2017 04 10;28(4):361-372. Epub 2017 Jan 10.

Departments of Pediatrics and Genetics, Stanford University , Stanford, California.

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http://dx.doi.org/10.1089/hum.2016.171DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5399734PMC
April 2017

Dieter C. Gruenert, PhD (1949-2016).

Nucleic Acid Ther 2016 08;26(4):266-7

4 The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine , iBio-Center for Urban Responses to Environmental Stressors, Detroit, Michigan.

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http://dx.doi.org/10.1089/nat.2016.29002.parDOI Listing
August 2016

Selecting the Best AAV Capsid for Human Studies.

Authors:
Mark A Kay

Mol Ther 2015 Dec;23(12):1800-1

Departments of Pediatrics and Genetics, Stanford University School of Medicine, Stanford, California, USA.

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http://dx.doi.org/10.1038/mt.2015.206DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4700120PMC
December 2015

Viral Vectors Take On HIV Infection.

N Engl J Med 2015 Aug;373(8):770-2

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http://dx.doi.org/10.1056/NEJMcibr1504232DOI Listing
August 2015

Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.

Hum Gene Ther Clin Dev 2015 Mar 12;26(1):5-14. Epub 2015 Feb 12.

1 Francis Owen Blood Research Laboratory, Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill , Chapel Hill, NC 27516.

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http://www.liebertpub.com/doi/10.1089/humc.2014.153
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http://dx.doi.org/10.1089/humc.2014.153DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4442577PMC
March 2015

RNA interference. Drugging RNAi.

Science 2015 Mar;347(6226):1069-70

Pediatrics and Genetics, Stanford University, Stanford, CA, USA.

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http://dx.doi.org/10.1126/science.1252967DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6018011PMC
March 2015

Novel codon-optimized mini-intronic plasmid for efficient, inexpensive, and xeno-free induction of pluripotency.

Sci Rep 2015 Jan 28;5:8081. Epub 2015 Jan 28.

1] Institute for Stem Cell Biology and Regenerative Medicine; Stanford University School of Medicine, Stanford, California 94305, USA [2] Stanford Cardiovascular Institute; Stanford University School of Medicine, Stanford, California 94305, USA [3] Department of Medicine, Division of Cardiology; Stanford University School of Medicine, Stanford, California 94305, USA.

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http://www.nature.com/articles/srep08081
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http://dx.doi.org/10.1038/srep08081DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4308704PMC
January 2015

Weak base pairing in both seed and 3' regions reduces RNAi off-targets and enhances si/shRNA designs.

Nucleic Acids Res 2014 Oct 30;42(19):12169-76. Epub 2014 Sep 30.

Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA

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http://dx.doi.org/10.1093/nar/gku854DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4231738PMC
October 2014

Regulation of microRNA-mediated gene silencing by microRNA precursors.

Nat Struct Mol Biol 2014 Sep 3;21(9):825-32. Epub 2014 Aug 3.

1] Department of Pediatrics, Stanford University, Stanford, California, USA. [2] Department of Genetics, Stanford University, Stanford, California, USA.

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http://dx.doi.org/10.1038/nsmb.2862DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4244528PMC
September 2014

Genome editing of isogenic human induced pluripotent stem cells recapitulates long QT phenotype for drug testing.

J Am Coll Cardiol 2014 Aug;64(5):451-9

Department of Medicine, Division of Cardiology, Stanford University School of Medicine, Stanford, California; Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, California; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, California. Electronic address:

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http://dx.doi.org/10.1016/j.jacc.2014.04.057DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4149735PMC
August 2014

Characterization of vector-based delivery of neurogenin-3 in murine diabetes.

Hum Gene Ther 2014 Jul 14;25(7):651-61. Epub 2014 Apr 14.

1 Departments of Pediatrics and Genetics, Stanford University , Stanford, CA 94305.

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http://dx.doi.org/10.1089/hum.2013.206DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4098120PMC
July 2014

Organ size control is dominant over Rb family inactivation to restrict proliferation in vivo.

Cell Rep 2014 Jul 10;8(2):371-81. Epub 2014 Jul 10.

Department of Pediatrics, Stanford University School of Medicine, 300 Pasteur Drive, Stanford, CA 94305, USA; Department of Genetics, Stanford University School of Medicine, 300 Pasteur Drive, Stanford, CA 94305, USA. Electronic address:

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http://dx.doi.org/10.1016/j.celrep.2014.06.025DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4128252PMC
July 2014

Recombinant AAV as a platform for translating the therapeutic potential of RNA interference.

Mol Ther 2014 Apr 19;22(4):692-701. Epub 2013 Dec 19.

1] Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA [2] Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA.

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http://dx.doi.org/10.1038/mt.2013.285DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3982504PMC
April 2014

Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.

Mol Ther 2014 Apr 6;22(4):725-33. Epub 2014 Jan 6.

Program in Epithelial Biology, Stanford University, School of Medicine, Stanford, California, USA.

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http://dx.doi.org/10.1038/mt.2013.290DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3982486PMC
April 2014

Engineering cellular resistance to HIV.

N Engl J Med 2014 Mar;370(10):968-9

From the Departments of Pediatrics and Genetics, Stanford University School of Medicine, Stanford, CA (M.A.K.); and the Ragon Institute of MGH, MIT, and Harvard, Cambridge, MA (B.D.W.).

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http://dx.doi.org/10.1056/NEJMe1400593DOI Listing
March 2014

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.

Nature 2014 Feb 25;506(7488):382-6. Epub 2013 Dec 25.

Stanford University, School of Medicine, Departments of Pediatrics and Genetics, 269 Campus Drive, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/nature12875DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3939040PMC
February 2014

The expanding repertoire of circular RNAs.

Mol Ther 2013 Jun;21(6):1112-4

Department of Pediatrics, Stanford University, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/mt.2013.101DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3677299PMC
June 2013

A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro.

Mol Ther 2013 May 5;21(5):954-63. Epub 2013 Mar 5.

Department of Pediatrics, Stanford University, Stanford, California 94305-5208, USA.

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http://linkinghub.elsevier.com/retrieve/pii/S152500161630675
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http://dx.doi.org/10.1038/mt.2013.33DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3666631PMC
May 2013

MicroRNAs in Cancer: the 22nd Hiroshima Cancer Seminar/the 4th Japanese Association for RNA Interference Joint International Symposium, 30 August 2012, Grand Prince Hotel Hiroshima.

Jpn J Clin Oncol 2013 May 13;43(5):579-82. Epub 2013 Mar 13.

Department of Cellular and Molecular Biology, Hiroshima University Graduate School of Biomedical Science, Hiroshima, Japan.

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http://dx.doi.org/10.1093/jjco/hyt037DOI Listing
May 2013

The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA.

Nucleic Acids Res 2013 Apr 8;41(6):3688-98. Epub 2013 Feb 8.

Department of Pediatrics, School of Medicine, Stanford University, 269 Pasteur Drive, Stanford, CA 94305, USA.

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http://dx.doi.org/10.1093/nar/gkt068DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3616702PMC
April 2013

Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level.

Mol Ther 2013 Jan 27;21(1):131-8. Epub 2012 Nov 27.

Department of Genetics, Stanford University School of Medicine, Stanford, California, USA.

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http://dx.doi.org/10.1038/mt.2012.244DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3538319PMC
January 2013

The extragenic spacer length between the 5' and 3' ends of the transgene expression cassette affects transgene silencing from plasmid-based vectors.

Mol Ther 2012 Nov 8;20(11):2111-9. Epub 2012 May 8.

Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/mt.2012.65DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3498813PMC
November 2012

rAAV-mediated tumorigenesis: still unresolved after an AAV assault.

Mol Ther 2012 Nov;20(11):2014-7

Departments of Pediatrics and Genetics, Stanford University, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/mt.2012.220DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3498811PMC
November 2012

Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression.

Mol Ther 2012 Oct 18;20(10):1912-23. Epub 2012 Sep 18.

Stanford University, Departments of Pediatrics and Genetics, Stanford, California 94305-5164, USA.

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http://dx.doi.org/10.1038/mt.2012.164DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3464642PMC
October 2012

AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence.

Mol Ther 2012 Oct 18;20(10):1902-11. Epub 2012 Sep 18.

Oregon Stem Cell Center, Papé Family Pediatric Research Institute, Oregon Health and Science University, Portland, Oregon 97239-3098, USA.

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http://dx.doi.org/10.1038/mt.2012.157DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3464636PMC
October 2012

Slicing-independent RISC activation requires the argonaute PAZ domain.

Curr Biol 2012 Aug 12;22(16):1536-42. Epub 2012 Jul 12.

Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.

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http://dx.doi.org/10.1016/j.cub.2012.06.040DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3604743PMC
August 2012

Expression determinants of mammalian argonaute proteins in mediating gene silencing.

Nucleic Acids Res 2012 Apr 30;40(8):3704-13. Epub 2011 Dec 30.

Department of Pediatrics, Stanford University, 269 Campus Drive CCSR 2110, Stanford, CA 94305, USA.

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http://dx.doi.org/10.1093/nar/gkr1274DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3333847PMC
April 2012

Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration.

J Clin Invest 2011 Dec 21;121(12):4850-60. Epub 2011 Nov 21.

Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, UCSF, San Francisco, California, USA.

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http://dx.doi.org/10.1172/JCI59261DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3226005PMC
December 2011

State-of-the-art gene-based therapies: the road ahead.

Authors:
Mark A Kay

Nat Rev Genet 2011 May 6;12(5):316-28. Epub 2011 Apr 6.

Department of Pediatrics and Genetics, Stanford University, 269 Campus Drive, Room 2105, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/nrg2971DOI Listing
May 2011

Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes.

Proc Natl Acad Sci U S A 2011 May 16;108(22):9208-13. Epub 2011 May 16.

Department of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.

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http://www.pnas.org/cgi/doi/10.1073/pnas.1018023108
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http://dx.doi.org/10.1073/pnas.1018023108DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3107324PMC
May 2011

Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors.

Nat Protoc 2011 Jan 23;6(1):78-88. Epub 2010 Dec 23.

Departments of Medicine and Radiology, Stanford University School of Medicine, Stanford, California, USA.

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http://dx.doi.org/10.1038/nprot.2010.173DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3657506PMC
January 2011

A robust system for production of minicircle DNA vectors.

Nat Biotechnol 2010 Dec 21;28(12):1287-9. Epub 2010 Nov 21.

Department of Pediatrics and Genetics, Stanford University School of Medicine, Stanford, California, USA.

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http://dx.doi.org/10.1038/nbt.1708DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4144359PMC
December 2010

Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.

Mol Ther 2010 Nov 17;18(11):1896-906. Epub 2010 Aug 17.

Max von Pettenkofer-Institut, Department of Virology, Ludwig-Maximilians-Universität Munich, Munich, Germany.

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http://dx.doi.org/10.1038/mt.2010.169DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2990515PMC
November 2010

FATP2 is a hepatic fatty acid transporter and peroxisomal very long-chain acyl-CoA synthetase.

Am J Physiol Endocrinol Metab 2010 Sep 8;299(3):E384-93. Epub 2010 Jun 8.

Department of Nutritional Sciences and Toxicology, University of California Berkeley, USA.

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http://dx.doi.org/10.1152/ajpendo.00226.2010DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2944282PMC
September 2010

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver.

J Clin Invest 2010 Sep 9;120(9):3106-19. Epub 2010 Aug 9.

Stanford University School of Medicine, Department of Pediatrics, Center for Clinical Sciences and Research, Stanford, California, USA.

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http://dx.doi.org/10.1172/JCI43565DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2929739PMC
September 2010

An in vitro-identified high-affinity nucleosome-positioning signal is capable of transiently positioning a nucleosome in vivo.

Epigenetics Chromatin 2010 Jul 1;3(1):13. Epub 2010 Jul 1.

Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA.

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http://dx.doi.org/10.1186/1756-8935-3-13DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2915997PMC
July 2010

How do miRNAs mediate translational repression?

Authors:
Shuo Gu Mark A Kay

Silence 2010 May 7;1(1):11. Epub 2010 May 7.

Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.

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http://dx.doi.org/10.1186/1758-907X-1-11DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2881910PMC
May 2010

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.

Hepatology 2010 Apr;51(4):1200-8

Oregon Stem Cell Center, Oregon Health and Science University, Portland, OR 97203, USA.

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http://dx.doi.org/10.1002/hep.23481DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3136243PMC
April 2010

Human tRNA-derived small RNAs in the global regulation of RNA silencing.

RNA 2010 Apr 24;16(4):673-95. Epub 2010 Feb 24.

Department of Pediatrics, Stanford University, Stanford, California 94305, USA.

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http://dx.doi.org/10.1261/rna.2000810DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2844617PMC
April 2010

A nonviral minicircle vector for deriving human iPS cells.

Nat Methods 2010 Mar 7;7(3):197-9. Epub 2010 Feb 7.

Departments of Medicine and Radiology, Stanford University School of Medicine, Stanford, California, USA.

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http://dx.doi.org/10.1038/nmeth.1426DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2892897PMC
March 2010

miR-122 continues to blaze the trail for microRNA therapeutics.

Mol Ther 2010 Feb;18(2):240-2

Department of Pediatrics and Genetics, Stanford University, Stanford, California, USA.

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http://dx.doi.org/10.1038/mt.2009.313DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2839286PMC
February 2010

Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice.

Mol Ther 2010 Jan 20;18(1):161-70. Epub 2009 Oct 20.

Department of Medicine, Division of Oncology, School of Medicine, Center for Clinical Sciences Research, Stanford University, Stanford, California 94305-5151, USA.

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http://dx.doi.org/10.1038/mt.2009.222DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2839214PMC
January 2010

Novel minicircle vector for gene therapy in murine myocardial infarction.

Circulation 2009 Sep;120(11 Suppl):S230-7

Department of Radiology, Stanford University School of Medicine, CA 94305-5344, USA.

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http://dx.doi.org/10.1161/CIRCULATIONAHA.108.841155DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3163107PMC
September 2009

RNAi and gene therapy: a mutual attraction.

Hematology Am Soc Hematol Educ Program 2007 :473-81

Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, CA 94305-5208, USA.

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http://dx.doi.org/10.1182/asheducation-2007.1.473DOI Listing
July 2009

A rapid protocol for construction and production of high-capacity adenoviral vectors.

Nat Protoc 2009 ;4(4):547-64

Department of Virology, Max von Pettenkofer-Institute, Ludwig-Maximilians University Munich, 80336 Munich, Germany.

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http://dx.doi.org/10.1038/nprot.2009.4DOI Listing
June 2009

Biological basis for restriction of microRNA targets to the 3' untranslated region in mammalian mRNAs.

Nat Struct Mol Biol 2009 Feb 1;16(2):144-50. Epub 2009 Feb 1.

The Center for Clinical Science Research, Room 2105, 269 Campus Drive, Stanford, California 94305-5164, USA.

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http://dx.doi.org/10.1038/nsmb.1552DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2713750PMC
February 2009

Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor.

J Cardiovasc Pharmacol 2009 Jan;53(1):18-23

Vecura, Clinical Research Center, Karolinska University Hospital Huddinge, Stockholm, Sweden.

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http://dx.doi.org/10.1097/FJC.0b013e318194234eDOI Listing
January 2009

Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic.

Mol Ther 2008 Sep 29;16(9):1630-6. Epub 2008 Jul 29.

Department of Pediatrics, Stanford University, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/mt.2008.144DOI Listing
September 2008

Capped small RNAs and MOV10 in human hepatitis delta virus replication.

Nat Struct Mol Biol 2008 Jul 15;15(7):714-21. Epub 2008 Jun 15.

Department of Pediatrics, Stanford University, 300 Pasteur Dr., Stanford, California 94305, USA.

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http://www.nature.com/articles/nsmb.1440
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http://dx.doi.org/10.1038/nsmb.1440DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2876191PMC
July 2008

In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses.

J Virol 2008 Jun 9;82(12):5887-911. Epub 2008 Apr 9.

Department of Pediatrics, School of Medicine, Stanford University, Stanford, CA 94305, USA.

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http://dx.doi.org/10.1128/JVI.00254-08DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2395137PMC
June 2008

Wandering eye for RNAi.

Nat Med 2008 Jun;14(6):611

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http://dx.doi.org/10.1038/nm0608-611DOI Listing
June 2008

Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation.

Mol Ther 2008 Mar 5;16(3):548-56. Epub 2008 Feb 5.

Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/sj.mt.6300399DOI Listing
March 2008

Prime time for small RNA-based therapeutics.

Authors:
Mark A Kay

Hum Gene Ther 2008 Jan;19(1):15-6

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http://dx.doi.org/10.1089/hum.2007.1220DOI Listing
January 2008

Postintegrative gene silencing within the Sleeping Beauty transposition system.

Mol Cell Biol 2007 Dec 15;27(24):8824-33. Epub 2007 Oct 15.

Stanford University School of Medicine, Department of Pediatrics, Stanford, CA 94305-5208, USA.

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http://dx.doi.org/10.1128/MCB.00498-07DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2169419PMC
December 2007

Cis-acting gene regulatory activities in the terminal regions of sleeping beauty DNA transposon-based vectors.

Hum Gene Ther 2007 Dec;18(12):1193-204

Department of Human Genetics, University of Aarhus, DK-8000 Aarhus, Denmark.

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http://dx.doi.org/10.1089/hum.2007.099DOI Listing
December 2007

Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?

J Clin Invest 2007 Dec;117(12):3633-41

Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA.

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http://content.the-jci.org/articles/view/34129
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http://dx.doi.org/10.1172/JCI34129DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2096424PMC
December 2007

Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency.

Mol Ther 2007 Nov 28;15(11):1955-62. Epub 2007 Aug 28.

Department of Pediatrics, Stanford University, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/sj.mt.6300293DOI Listing
November 2007

DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice.

J Virol 2007 Oct 8;81(20):11290-303. Epub 2007 Aug 8.

Department of Molecular Genetics & Biochemistry, University of Pittsburgh School of Medicine, W1244 BSTWR, 200 Lothrop St., Pittsburgh, PA 15261, USA.

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http://dx.doi.org/10.1128/JVI.00963-07DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2045527PMC
October 2007

The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice.

J Virol 2007 Oct 8;81(20):11304-21. Epub 2007 Aug 8.

Department of Molecular Genetics & Biochemistry, University of Pittsburgh School of Medicine, W1244 BSTWR, 200 Lothrop St., Pittsburgh, PA 15261, USA.

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http://dx.doi.org/10.1128/JVI.01225-07DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2045570PMC
October 2007

Rapid and stable knockdown of an endogenous gene in retinal pigment epithelium.

Hum Gene Ther 2007 Oct;18(10):871-80

Beckman Vision Center, University of California, San Francisco, San Francisco, CA 94143, USA.

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http://dx.doi.org/10.1089/hum.2007.065DOI Listing
October 2007

AAV vectors and tumorigenicity.

Authors:
Mark A Kay

Nat Biotechnol 2007 Oct;25(10):1111-3

Department of Pediatrics, Rm G305, Stanford University, Stanford, California 94305, USA.

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http://dx.doi.org/10.1038/nbt1007-1111DOI Listing
October 2007

Distinct pathways of genomic progression to benign and malignant tumors of the liver.

Proc Natl Acad Sci U S A 2007 Sep 4;104(37):14771-6. Epub 2007 Sep 4.

G. W. Hooper Foundation and Department of Microbiology and Immunology, University of California, San Francisco, CA 94143, USA.

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http://dx.doi.org/10.1073/pnas.0706578104DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1964540PMC
September 2007

Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice.

Nat Biotechnol 2007 Aug 29;25(8):903-10. Epub 2007 Jul 29.

Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon 97239, USA.

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http://dx.doi.org/10.1038/nbt1326DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3404624PMC
August 2007

Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery.

Mol Ther 2007 Jul 24;15(7):1273-9. Epub 2007 Apr 24.

University of Minnesota Cancer Center, Department of Pediatrics, Division of Hematology-Oncology, Blood and Marrow Transplantation, University of Minnesota, Minneapolis, USA.

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http://linkinghub.elsevier.com/retrieve/pii/S152500161632413
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http://dx.doi.org/10.1038/sj.mt.6300178DOI Listing
July 2007

Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo.

Mol Ther 2007 Jul 24;15(7):1348-55. Epub 2007 Apr 24.

Department of Pediatrics, Stanford University School of Medicine, Stanford, California, USA.

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http://dx.doi.org/10.1038/sj.mt.6300177DOI Listing
July 2007

Site-directed transposon integration in human cells.

Nucleic Acids Res 2007 7;35(7):e50. Epub 2007 Mar 7.

Department of Pediatrics and Genetics, Stanford University School of Medicine, Stanford, CA 94305-5208, USA.

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http://dx.doi.org/10.1093/nar/gkm089DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1874657PMC
June 2007

Combinatorial RNAi: a winning strategy for the race against evolving targets?

Mol Ther 2007 May 20;15(5):878-88. Epub 2007 Feb 20.

Department of Pediatrics, Stanford University School of Medicine, Stanford, CA 94305, USA.

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http://dx.doi.org/10.1038/sj.mt.6300116DOI Listing
May 2007