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Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases.

De Caneva A, Porro F, Bortolussi G, Sola R, Lisjak M, Barzel A, Giacca M, Kay MA, Vlahoviček K, Zentilin L, Muro AF.

JCI Insight. 2019 Jun 18;5. pii: 128863. doi: 10.1172/jci.insight.128863.


An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging.

Oh Y, Park Y, Cho JH, Wu H, Paulk NK, Liu LX, Kim N, Kay MA, Wu JC, Lin MZ.

Nat Chem Biol. 2019 May;15(5):433-436. doi: 10.1038/s41589-019-0256-z. Epub 2019 Apr 1.


miR-122 removal in the liver activates imprinted microRNAs and enables more effective microRNA-mediated gene repression.

Valdmanis PN, Kim HK, Chu K, Zhang F, Xu J, Munding EM, Shen J, Kay MA.

Nat Commun. 2018 Dec 14;9(1):5321. doi: 10.1038/s41467-018-07786-7.


Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle.

Paulk NK, Pekrun K, Charville GW, Maguire-Nguyen K, Wosczyna MN, Xu J, Zhang Y, Lisowski L, Yoo B, Vilches-Moure JG, Lee GK, Shrager JB, Rando TA, Kay MA.

Mol Ther Methods Clin Dev. 2018 Jul 24;10:144-155. doi: 10.1016/j.omtm.2018.06.001. eCollection 2018 Sep 21.


Discovery of {4-[4,9-bis(ethyloxy)-1-oxo-1,3-dihydro-2H-benzo[f]isoindol-2-yl]-2-fluorophenyl}acetic acid (GSK726701A), a novel EP4 receptor partial agonist for the treatment of pain.

Healy MP, Allan AC, Bailey K, Billinton A, Chessell IP, Clayton NM, Giblin GMP, Kay MA, Khaznadar T, Michel AD, Naylor A, Price H, Spalding DJ, Stevens DA, Swarbrick ME, Wilson AW.

Bioorg Med Chem Lett. 2018 Jun 1;28(10):1892-1896. doi: 10.1016/j.bmcl.2018.03.091. Epub 2018 Mar 31.


Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity.

Winters IP, Chiou SH, Paulk NK, McFarland CD, Lalgudi PV, Ma RK, Lisowski L, Connolly AJ, Petrov DA, Kay MA, Winslow MM.

Nat Commun. 2017 Dec 12;8(1):2053. doi: 10.1038/s41467-017-01519-y.


Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaam6375. doi: 10.1126/scitranslmed.aam6375.


A transfer-RNA-derived small RNA regulates ribosome biogenesis.

Kim HK, Fuchs G, Wang S, Wei W, Zhang Y, Park H, Roy-Chaudhuri B, Li P, Xu J, Chu K, Zhang F, Chua MS, So S, Zhang QC, Sarnow P, Kay MA.

Nature. 2017 Dec 7;552(7683):57-62. doi: 10.1038/nature25005. Epub 2017 Nov 29.


Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA.

Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25.


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

Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C.

Mol Ther. 2017 Nov 1;25(11):2477-2489. doi: 10.1016/j.ymthe.2017.09.020. Epub 2017 Sep 25.


Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.

Porro F, Bortolussi G, Barzel A, De Caneva A, Iaconcig A, Vodret S, Zentilin L, Kay MA, Muro AF.

EMBO Mol Med. 2017 Oct;9(10):1346-1355. doi: 10.15252/emmm.201707601.


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

Lu J, Zhang F, Fire AZ, Kay MA.

Mol Ther. 2017 May 3;25(5):1187-1198. doi: 10.1016/j.ymthe.2017.03.003. Epub 2017 Mar 30.


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

Valdmanis PN, Kay MA.

Hum Gene Ther. 2017 Apr;28(4):361-372. doi: 10.1089/hum.2016.171. Epub 2017 Jan 10.


Regulated complex assembly safeguards the fidelity of Sleeping Beauty transposition.

Wang Y, Pryputniewicz-Dobrinska D, Nagy EÉ, Kaufman CD, Singh M, Yant S, Wang J, Dalda A, Kay MA, Ivics Z, Izsvák Z.

Nucleic Acids Res. 2017 Jan 9;45(1):311-326. doi: 10.1093/nar/gkw1164. Epub 2016 Dec 1.


A 5' Noncoding Exon Containing Engineered Intron Enhances Transgene Expression from Recombinant AAV Vectors in vivo.

Lu J, Williams JA, Luke J, Zhang F, Chu K, Kay MA.

Hum Gene Ther. 2017 Jan;28(1):125-134. doi: 10.1089/hum.2016.140.


Increased precursor microRNA-21 following status epilepticus can compete with mature microRNA-21 to alter translation.

Chak K, Roy-Chaudhuri B, Kim HK, Kemp KC, Porter BE, Kay MA.

Exp Neurol. 2016 Dec;286:137-146. doi: 10.1016/j.expneurol.2016.10.003. Epub 2016 Oct 8.


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

Disterer P, Kay MA, Parker GC.

Nucleic Acid Ther. 2016 Aug;26(4):266-7. doi: 10.1089/nat.2016.29002.par. No abstract available.


A universal system to select gene-modified hepatocytes in vivo.

Nygaard S, Barzel A, Haft A, Major A, Finegold M, Kay MA, Grompe M.

Sci Transl Med. 2016 Jun 8;8(342):342ra79. doi: 10.1126/scitranslmed.aad8166.


A bright cyan-excitable orange fluorescent protein facilitates dual-emission microscopy and enhances bioluminescence imaging in vivo.

Chu J, Oh Y, Sens A, Ataie N, Dana H, Macklin JJ, Laviv T, Welf ES, Dean KM, Zhang F, Kim BB, Tang CT, Hu M, Baird MA, Davidson MW, Kay MA, Fiolka R, Yasuda R, Kim DS, Ng HL, Lin MZ.

Nat Biotechnol. 2016 Jul;34(7):760-7. doi: 10.1038/nbt.3550. Epub 2016 May 30.


A Tribute to George Stamatoyannopoulos.

Srivastava A, Kay MA, Athanasopoulos T, Angastiniotis M, Anagnostopoulos A, Karponi G, Yannaki E, Zon LI, Lederer CW, Phylactides MS, Kleanthous M.

Hum Gene Ther. 2016 Apr;27(4):280-6. doi: 10.1089/hum.2016.29025.gst. No abstract available.


RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice.

Valdmanis PN, Gu S, Chu K, Jin L, Zhang F, Munding EM, Zhang Y, Huang Y, Kutay H, Ghoshal K, Lisowski L, Kay MA.

Nat Med. 2016 May;22(5):557-62. doi: 10.1038/nm.4079. Epub 2016 Apr 11.


Selecting the Best AAV Capsid for Human Studies.

Kay MA.

Mol Ther. 2015 Dec;23(12):1800-1. doi: 10.1038/mt.2015.206. No abstract available.


Viral Vectors Take On HIV Infection.

Mellins ED, Kay MA.

N Engl J Med. 2015 Aug 20;373(8):770-2. doi: 10.1056/NEJMcibr1504232. No abstract available.


RNA interference. Drugging RNAi.

Haussecker D, Kay MA.

Science. 2015 Mar 6;347(6226):1069-70. doi: 10.1126/science.1252967. No abstract available.


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

Nichols TC, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):5-14. doi: 10.1089/humc.2014.153. Epub 2015 Feb 12. Review.


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

Diecke S, Lu J, Lee J, Termglinchan V, Kooreman NG, Burridge PW, Ebert AD, Churko JM, Sharma A, Kay MA, Wu JC.

Sci Rep. 2015 Jan 28;5:8081. doi: 10.1038/srep08081.


Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa.

Sebastiano V, Zhen HH, Haddad B, Bashkirova E, Melo SP, Wang P, Leung TL, Siprashvili Z, Tichy A, Li J, Ameen M, Hawkins J, Lee S, Li L, Schwertschkow A, Bauer G, Lisowski L, Kay MA, Kim SK, Lane AT, Wernig M, Oro AE.

Sci Transl Med. 2014 Nov 26;6(264):264ra163. doi: 10.1126/scitranslmed.3009540. Erratum in: Sci Transl Med. 2014 Dec 17;6(267):267er8. Derafshi, Bahareh Haddad [corrected to Haddad, Bahareh].


Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM.

N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309.


Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM, Kay MA.

Nature. 2015 Jan 15;517(7534):360-4. doi: 10.1038/nature13864. Epub 2014 Oct 29.


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

Gu S, Zhang Y, Jin L, Huang Y, Zhang F, Bassik MC, Kampmann M, Kay MA.

Nucleic Acids Res. 2014 Oct 29;42(19):12169-76. doi: 10.1093/nar/gku854. Epub 2014 Sep 30.


Perceptions of barriers and facilitators to health behavior change among veteran cancer survivors.

Beehler GP, Rodrigues AE, Kay MA, Kiviniemi MT, Steinbrenner L.

Mil Med. 2014 Sep;179(9):998-1005. doi: 10.7205/MILMED-D-14-00027.


Regulation of microRNA-mediated gene silencing by microRNA precursors.

Roy-Chaudhuri B, Valdmanis PN, Zhang Y, Wang Q, Luo QJ, Kay MA.

Nat Struct Mol Biol. 2014 Sep;21(9):825-32. doi: 10.1038/nsmb.2862. Epub 2014 Aug 3.


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

Wang Y, Liang P, Lan F, Wu H, Lisowski L, Gu M, Hu S, Kay MA, Urnov FD, Shinnawi R, Gold JD, Gepstein L, Wu JC.

J Am Coll Cardiol. 2014 Aug 5;64(5):451-9. doi: 10.1016/j.jacc.2014.04.057.


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

Ehmer U, Zmoos AF, Auerbach RK, Vaka D, Butte AJ, Kay MA, Sage J.

Cell Rep. 2014 Jul 24;8(2):371-81. doi: 10.1016/j.celrep.2014.06.025. Epub 2014 Jul 10.


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

Phillips N, Kay MA.

Hum Gene Ther. 2014 Jul;25(7):651-61. doi: 10.1089/hum.2013.206. Epub 2014 Apr 14.


Engineering cellular resistance to HIV.

Kay MA, Walker BD.

N Engl J Med. 2014 Mar 6;370(10):968-9. doi: 10.1056/NEJMe1400593. No abstract available.


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

Lisowski L, Dane AP, Chu K, Zhang Y, Cunningham SC, Wilson EM, Nygaard S, Grompe M, Alexander IE, Kay MA.

Nature. 2014 Feb 20;506(7488):382-6. doi: 10.1038/nature12875. Epub 2013 Dec 25.


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

Melo SP, Lisowski L, Bashkirova E, Zhen HH, Chu K, Keene DR, Marinkovich MP, Kay MA, Oro AE.

Mol Ther. 2014 Apr;22(4):725-33. doi: 10.1038/mt.2013.290. Epub 2014 Jan 6.


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

Borel F, Kay MA, Mueller C.

Mol Ther. 2014 Apr;22(4):692-701. doi: 10.1038/mt.2013.285. Epub 2013 Dec 19. Review.


Upregulation of the microRNA cluster at the Dlk1-Dio3 locus in lung adenocarcinoma.

Valdmanis PN, Roy-Chaudhuri B, Kim HK, Sayles LC, Zheng Y, Chuang CH, Caswell DR, Chu K, Zhang Y, Winslow MM, Sweet-Cordero EA, Kay MA.

Oncogene. 2015 Jan 2;34(1):94-103. doi: 10.1038/onc.2013.523. Epub 2013 Dec 9.


Lasting impact: understanding the psychosocial implications of cancer among military veterans.

Beehler GP, Rodrigues AE, Kay MA, Kiviniemi MT, Steinbrenner L.

J Psychosoc Oncol. 2013;31(4):430-50. doi: 10.1080/07347332.2013.798762.


The expanding repertoire of circular RNAs.

Valdmanis PN, Kay MA.

Mol Ther. 2013 Jun;21(6):1112-4. doi: 10.1038/mt.2013.101. No abstract available.


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.

Tahara H, Kay MA, Yasui W, Tahara E.

Jpn J Clin Oncol. 2013 May;43(5):579-82. doi: 10.1093/jjco/hyt037. Epub 2013 Mar 13.


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

Lu J, Zhang F, Kay MA.

Mol Ther. 2013 May;21(5):954-63. doi: 10.1038/mt.2013.33. Epub 2013 Mar 5.


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

Lisowski L, Elazar M, Chu K, Glenn JS, Kay MA.

Nucleic Acids Res. 2013 Apr 1;41(6):3688-98. doi: 10.1093/nar/gkt068. Epub 2013 Feb 8.


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

Gracey Maniar LE, Maniar JM, Chen ZY, Lu J, Fire AZ, Kay MA.

Mol Ther. 2013 Jan;21(1):131-8. doi: 10.1038/mt.2012.244. Epub 2012 Nov 27.


The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo.

Gu S, Jin L, Zhang Y, Huang Y, Zhang F, Valdmanis PN, Kay MA.

Cell. 2012 Nov 9;151(4):900-911. doi: 10.1016/j.cell.2012.09.042.


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

Valdmanis PN, Lisowski L, Kay MA.

Mol Ther. 2012 Nov;20(11):2014-7. doi: 10.1038/mt.2012.220. No abstract available.


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

Wang Z, Lisowski L, Finegold MJ, Nakai H, Kay MA, Grompe M.

Mol Ther. 2012 Oct;20(10):1902-11. doi: 10.1038/mt.2012.157. Epub 2012 Sep 18.


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

Lisowski L, Lau A, Wang Z, Zhang Y, Zhang F, Grompe M, Kay MA.

Mol Ther. 2012 Oct;20(10):1912-23. doi: 10.1038/mt.2012.164. Epub 2012 Sep 18.

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