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Items: 12

1.

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.

Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A.

Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2.

PMID:
31375812
2.

Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.

Gruntman AM, Gernoux G, Tang Q, Ye GJ, Knop DR, Wang G, Benson J, Coleman KE, Keeler AM, Mueller C, Chicoine LG, Chulay JD, Flotte TR.

Mol Ther Methods Clin Dev. 2019 Feb 2;13:233-242. doi: 10.1016/j.omtm.2019.01.013. eCollection 2019 Jun 14.

3.

Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity.

Guilbaud M, Devaux M, Couzini├ę C, Le Duff J, Toromanoff A, Vandamme C, Jaulin N, Gernoux G, Larcher T, Moullier P, Le Guiner C, Adjali O.

Hum Gene Ther. 2019 Jul;30(7):802-813. doi: 10.1089/hum.2018.234. Epub 2019 Apr 16.

4.

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques.

Borel F, Gernoux G, Sun H, Stock R, Blackwood M, Brown RH Jr, Mueller C.

Sci Transl Med. 2018 Oct 31;10(465). pii: eaau6414. doi: 10.1126/scitranslmed.aau6414.

PMID:
30381409
5.

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.

Pfister EL, DiNardo N, Mondo E, Borel F, Conroy F, Fraser C, Gernoux G, Han X, Hu D, Johnson E, Kennington L, Liu P, Reid SJ, Sapp E, Vodicka P, Kuchel T, Morton AJ, Howland D, Moser R, Sena-Esteves M, Gao G, Mueller C, DiFiglia M, Aronin N.

Hum Gene Ther. 2018 Jun;29(6):663-673. doi: 10.1089/hum.2017.199. Epub 2018 Feb 23.

PMID:
29207890
6.

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of ╬▒-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.

7.

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.

Mueller C, Gernoux G, Gruntman AM, Borel F, Reeves EP, Calcedo R, Rouhani FN, Yachnis A, Humphries M, Campbell-Thompson M, Messina L, Chulay JD, Trapnell B, Wilson JM, McElvaney NG, Flotte TR.

Mol Ther. 2017 Jun 7;25(6):1387-1394. doi: 10.1016/j.ymthe.2017.03.029. Epub 2017 Apr 10.

8.

Regulatory and Exhausted T Cell Responses to AAV Capsid.

Gernoux G, Wilson JM, Mueller C.

Hum Gene Ther. 2017 Apr;28(4):338-349. doi: 10.1089/hum.2017.022.

9.

Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.

Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabrera GC, Song L, Su Q, Gao GP, Elmallah MK, Brown RH Jr, Mueller C.

Hum Gene Ther. 2016 Jan;27(1):19-31. doi: 10.1089/hum.2015.122. Erratum in: Hum Gene Ther. 2016 Aug;27(8):652. Hum Gene Ther. 2016 Dec;27(12 ):1015.

10.

Early interaction of adeno-associated virus serotype 8 vector with the host immune system following intramuscular delivery results in weak but detectable lymphocyte and dendritic cell transduction.

Gernoux G, Guilbaud M, Dubreil L, Larcher T, Babarit C, Ledevin M, Jaulin N, Planel P, Moullier P, Adjali O.

Hum Gene Ther. 2015 Jan;26(1):1-13. doi: 10.1089/hum.2014.070.

PMID:
25333770
11.

Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.

Chuah MK, Petrus I, De Bleser P, Le Guiner C, Gernoux G, Adjali O, Nair N, Willems J, Evens H, Rincon MY, Matrai J, Di Matteo M, Samara-Kuko E, Yan B, Acosta-Sanchez A, Meliani A, Cherel G, Blouin V, Christophe O, Moullier P, Mingozzi F, VandenDriessche T.

Mol Ther. 2014 Sep;22(9):1605-13. doi: 10.1038/mt.2014.114. Epub 2014 Jun 23.

12.

Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping.

Ni W, Le Guiner C, Gernoux G, Penaud-Budloo M, Moullier P, Snyder RO.

Gene Ther. 2011 Jul;18(7):709-18. doi: 10.1038/gt.2011.19. Epub 2011 Mar 10.

PMID:
21390073

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