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Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine EF, Genetti CA, Dies K, Barton B, Williams L, Goodlett BD, Riley BL, Pasternak A, Berry ER, Pflock KA, Chu S, Reed C, Tyndall K, Agrawal PB, Beggs AH, Grant PE, Urion DK, Snyder RO, Waisbren SE, Poduri A, Park PJ, Patterson A, Biffi A, Mazzulli JR, Bodamer O, Berde CB, Yu TW.

N Engl J Med. 2019 Oct 24;381(17):1644-1652. doi: 10.1056/NEJMoa1813279. Epub 2019 Oct 9.


Stability of the adeno-associated virus 8 reference standard material.

Penaud-Budloo M, Broucque F, Harrouet K, Bouzelha M, Saleun S, Douthe S, D'Costa S, Ogram S, Adjali O, Blouin V, Lock M, Snyder RO, Ayuso E.

Gene Ther. 2019 May;26(5):211-215. doi: 10.1038/s41434-019-0072-9. Epub 2019 Mar 29.


Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines.

Kotin RM, Snyder RO.

Hum Gene Ther. 2017 Apr;28(4):350-360. doi: 10.1089/hum.2017.042.


Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR.

D'Costa S, Blouin V, Broucque F, Penaud-Budloo M, François A, Perez IC, Le Bec C, Moullier P, Snyder RO, Ayuso E.

Mol Ther Methods Clin Dev. 2016 Mar 30;5:16019. doi: 10.1038/mtm.2016.19. eCollection 2016.


Establishment of two quantitative nested qPCR assays targeting the human EPO transgene.

Neuberger EW, Perez I, Le Guiner C, Moser D, Ehlert T, Allais M, Moullier P, Simon P, Snyder RO.

Gene Ther. 2016 Apr;23(4):330-9. doi: 10.1038/gt.2016.2. Epub 2016 Jan 11.


Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA.

Dupont JB, Tournaire B, Georger C, Marolleau B, Jeanson-Leh L, Ledevin M, Lindenbaum P, Lecomte E, Cogné B, Dubreil L, Larcher T, Gjata B, Van Wittenberghe L, Le Guiner C, Penaud-Budloo M, Snyder RO, Moullier P, Léger A.

Mol Ther Methods Clin Dev. 2015 Apr 8;2:15010. doi: 10.1038/mtm.2015.10. eCollection 2015.


Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.

Ayuso E, Blouin V, Lock M, McGorray S, Leon X, Alvira MR, Auricchio A, Bucher S, Chtarto A, Clark KR, Darmon C, Doria M, Fountain W, Gao G, Gao K, Giacca M, Kleinschmidt J, Leuchs B, Melas C, Mizukami H, Müller M, Noordman Y, Bockstael O, Ozawa K, Pythoud C, Sumaroka M, Surosky R, Tenenbaum L, van der Linden I, Weins B, Wright JF, Zhang X, Zentilin L, Bosch F, Snyder RO, Moullier P.

Hum Gene Ther. 2014 Nov;25(11):977-87. doi: 10.1089/hum.2014.057.


PCR-based detection of gene transfer vectors: application to gene doping surveillance.

Perez IC, Le Guiner C, Ni W, Lyles J, Moullier P, Snyder RO.

Anal Bioanal Chem. 2013 Dec;405(30):9641-53. doi: 10.1007/s00216-013-7264-8. Epub 2013 Aug 4. Review.


Gene therapy for aromatic L-amino acid decarboxylase deficiency.

Hwu WL, Muramatsu S, Tseng SH, Tzen KY, Lee NC, Chien YH, Snyder RO, Byrne BJ, Tai CH, Wu RM.

Sci Transl Med. 2012 May 16;4(134):134ra61. doi: 10.1126/scitranslmed.3003640.


Development and utility of an internal threshold control (ITC) real-time PCR assay for exogenous DNA detection.

Ni W, Le Guiner C, Moullier P, Snyder RO.

PLoS One. 2012;7(5):e36461. doi: 10.1371/journal.pone.0036461. Epub 2012 May 3.


Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver.

Nowrouzi A, Penaud-Budloo M, Kaeppel C, Appelt U, Le Guiner C, Moullier P, von Kalle C, Snyder RO, Schmidt M.

Mol Ther. 2012 Jun;20(6):1177-86. doi: 10.1038/mt.2012.47. Epub 2012 Mar 27.


Recombinant adeno-associated viral vector reference standards.

Moullier P, Snyder RO.

Methods Enzymol. 2012;507:297-311. doi: 10.1016/B978-0-12-386509-0.00015-6.


rAAV vector product characterization and stability studies.

Snyder RO, Audit M, Francis JD.

Methods Mol Biol. 2011;807:405-28. doi: 10.1007/978-1-61779-370-7_17.


Effects of transient overexpression or knockdown of cytochrome P450 reductase on reactive oxygen species generation and hypoxia reoxygenation injury in liver cells.

Pillai VC, Snyder RO, Gumaste U, Thekkumkara TJ, Mehvar R.

Clin Exp Pharmacol Physiol. 2011 Dec;38(12):846-53. doi: 10.1111/j.1440-1681.2011.05622.x.


Adeno-associated viral vector-mediated transgene expression is independent of DNA methylation in primate liver and skeletal muscle.

Léger A, Le Guiner C, Nickerson ML, McGee Im K, Ferry N, Moullier P, Snyder RO, Penaud-Budloo M.

PLoS One. 2011;6(6):e20881. doi: 10.1371/journal.pone.0020881. Epub 2011 Jun 8.


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.


Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material.

Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, Chiorini JA, Chtarto A, Clark KR, Conlon T, Darmon C, Doria M, Douar A, Flotte TR, Francis JD, Francois A, Giacca M, Korn MT, Korytov I, Leon X, Leuchs B, Lux G, Melas C, Mizukami H, Moullier P, Müller M, Ozawa K, Philipsberg T, Poulard K, Raupp C, Rivière C, Roosendaal SD, Samulski RJ, Soltys SM, Surosky R, Tenenbaum L, Thomas DL, van Montfort B, Veres G, Wright JF, Xu Y, Zelenaia O, Zentilin L, Snyder RO.

Hum Gene Ther. 2010 Oct;21(10):1273-85. doi: 10.1089/hum.2009.223.


Role of cyclic AMP-dependent kinase response element-binding protein in recombinant adeno-associated virus-mediated transduction of heart muscle cells.

Dean J, Plante J, Huggins GS, Snyder RO, Aikawa R.

Hum Gene Ther. 2009 Sep;20(9):1005-12. doi: 10.1089/hum.2009.054.


Adeno-associated virus capsid serotype identification: Analytical methods development and application.

Van Vliet K, Mohiuddin Y, McClung S, Blouin V, Rolling F, Moullier P, Agbandje-McKenna M, Snyder RO.

J Virol Methods. 2009 Aug;159(2):167-77. doi: 10.1016/j.jviromet.2009.03.020. Epub 2009 Mar 26.


Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle.

Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, Le Guiner C.

Mol Ther. 2008 Jul;16(7):1291-1299. doi: 10.1038/mt.2008.87. Epub 2016 Dec 8.


International efforts for recombinant adeno-associated viral vector reference standards.

Moullier P, Snyder RO.

Mol Ther. 2008 Jul;16(7):1185-8. doi: 10.1038/mt.2008.125. No abstract available.


Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle.

Penaud-Budloo M, Le Guiner C, Nowrouzi A, Toromanoff A, Chérel Y, Chenuaud P, Schmidt M, von Kalle C, Rolling F, Moullier P, Snyder RO.

J Virol. 2008 Aug;82(16):7875-85. doi: 10.1128/JVI.00649-08. Epub 2008 Jun 4.


Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle.

Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, Le Guiner C.

Mol Ther. 2008 Jul;16(7):1291-9. doi: 10.1038/mt.2008.87. Epub 2008 May 6.


The role of the adeno-associated virus capsid in gene transfer.

Van Vliet KM, Blouin V, Brument N, Agbandje-McKenna M, Snyder RO.

Methods Mol Biol. 2008;437:51-91. doi: 10.1007/978-1-59745-210-6_2. Review.


Immune responses to gene product of inducible promoters.

Le Guiner C, Stieger K, Snyder RO, Rolling F, Moullier P.

Curr Gene Ther. 2007 Oct;7(5):334-46. Review.


Viral vectors for in vivo gene transfer in Parkinson's disease: properties and clinical grade production.

Mandel RJ, Burger C, Snyder RO.

Exp Neurol. 2008 Jan;209(1):58-71. Epub 2007 Aug 24. Review.


Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults.

Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A, Garlington W, Baker D, Song S, Berns KI, Muzyczka N, Snyder RO, Byrne BJ, Flotte TR.

Hum Gene Ther. 2006 Dec;17(12):1177-86.


Proteolytic mapping of the adeno-associated virus capsid.

Van Vliet K, Blouin V, Agbandje-McKenna M, Snyder RO.

Mol Ther. 2006 Dec;14(6):809-21. Epub 2006 Sep 27.


Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system.

Kohlbrenner E, Aslanidi G, Nash K, Shklyaev S, Campbell-Thompson M, Byrne BJ, Snyder RO, Muzyczka N, Warrington KH Jr, Zolotukhin S.

Mol Ther. 2005 Dec;12(6):1217-25. Epub 2005 Oct 6.


Adeno-associated viral vectors for clinical gene transfer studies.

Snyder RO, Francis J.

Curr Gene Ther. 2005 Jun;5(3):311-21. Review.


Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors.

Mohiuddin I, Loiler S, Zolotukhin I, Byrne BJ, Flotte TR, Snyder RO.

Mol Ther. 2005 Feb;11(2):320-6.


Development and characterization of a cell line for large-scale, serum-free production of recombinant adeno-associated viral vectors.

Farson D, Harding TC, Tao L, Liu J, Powell S, Vimal V, Yendluri S, Koprivnikar K, Ho K, Twitty C, Husak P, Lin A, Snyder RO, Donahue BA.

J Gene Med. 2004 Dec;6(12):1369-81.


Immunity to adeno-associated virus serotype 2 delivered transgenes imparted by genetic predisposition to autoimmunity.

Zhang YC, Powers M, Wasserfall C, Brusko T, Song S, Flotte T, Snyder RO, Potter M, Scott-Jorgensen M, Campbell-Thompson M, Crawford JM, Nick HS, Agarwal A, Ellis TM, Atkinson MA.

Gene Ther. 2004 Feb;11(3):233-40.


Production of clinical-grade recombinant adeno-associated virus vectors.

Snyder RO, Flotte TR.

Curr Opin Biotechnol. 2002 Oct;13(5):418-23. Review.


Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors.

Zolotukhin S, Potter M, Zolotukhin I, Sakai Y, Loiler S, Fraites TJ Jr, Chiodo VA, Phillipsberg T, Muzyczka N, Hauswirth WW, Flotte TR, Byrne BJ, Snyder RO.

Methods. 2002 Oct;28(2):158-67.


Cardiomyocyte-specific gene expression following recombinant adeno-associated viral vector transduction.

Aikawa R, Huggins GS, Snyder RO.

J Biol Chem. 2002 May 24;277(21):18979-85. Epub 2002 Mar 11.


Recombinant adeno-associated viral vector production using stable packaging and producer cell lines.

Mathews LC, Gray JT, Gallagher MR, Snyder RO.

Methods Enzymol. 2002;346:393-413. No abstract available.


AAV and RT-PCR: true or false?

Snyder RO.

Mol Ther. 2000 May;1(5 Pt 1):389-90. Review. No abstract available.


Gene delivery to in situ veins: differential effects of adenovirus and adeno-associated viral vectors.

Eslami MH, Gangadharan SP, Sui X, Rhynhart KK, Snyder RO, Conte MS.

J Vasc Surg. 2000 Jun;31(6):1149-59.


Selective uptake and sustained expression of AAV vectors following subcutaneous delivery.

Donahue BA, McArthur JG, Spratt SK, Bohl D, Lagarde C, Sanchez L, Kaspar BA, Sloan BA, Lee YL, Danos O, Snyder RO.

J Gene Med. 1999 Jan-Feb;1(1):31-42.


Adeno-associated virus-mediated gene delivery.

Snyder RO.

J Gene Med. 1999 May-Jun;1(3):166-75. Review.


Epitope mapping of human anti-adeno-associated virus type 2 neutralizing antibodies: implications for gene therapy and virus structure.

Moskalenko M, Chen L, van Roey M, Donahue BA, Snyder RO, McArthur JG, Patel SD.

J Virol. 2000 Feb;74(4):1761-6.


Viral gene delivery selectively restores feeding and prevents lethality of dopamine-deficient mice.

Szczypka MS, Mandel RJ, Donahue BA, Snyder RO, Leff SE, Palmiter RD.

Neuron. 1999 Jan;22(1):167-78.


Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle.

Vincent-Lacaze N, Snyder RO, Gluzman R, Bohl D, Lagarde C, Danos O.

J Virol. 1999 Mar;73(3):1949-55.


Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors.

Snyder RO, Miao C, Meuse L, Tubb J, Donahue BA, Lin HF, Stafford DW, Patel S, Thompson AR, Nichols T, Read MS, Bellinger DA, Brinkhous KM, Kay MA.

Nat Med. 1999 Jan;5(1):64-70.


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