Valdmanis PN, Kay MA. Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond. Human Gene Therapy. 2017 Apr;28(4):361-372.
Valdmanis PN, Gu S, Chu K, Jin L, Zhang F, Munding EM, Huang Y, Kutay H, Ghoshal K, Lisowski L, Kay MA. RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice. Nature Medicine 2016 May;22(5)557-62.
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. Upregulation of the microRNA cluster at the Dlk1-Dio3 locus in lung adenocarcinoma. Oncogene. 2015 Jan 2;34(1):94-103.
Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector, LP, Porteus MH, Gaensler KM, Kay MA. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature 2015 Jan 15;517(7534):360-4.
Roy-Chaudhuri B, Valdmanis PN, Zhang Y, Wang Q, Luo QJ, Kay MA. Regulation of microRNA-mediated gene silencing by microRNA precursors. Nature Structural & Molecular Biology 2014 Sep;21(9):825-32.
Gu S, Jin L, Zhang Y, Huang Y, Zhang F, Valdmanis PN, Kay MA. The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo. Cell 2012 Nov 9;151(4):900-11.
Valdmanis PN, Gu S, Schüermann N, Sethupathy P, Grimm D, Kay MA. Expression determinants of mammalian argonaute proteins in mediating gene silencing. Nucleic Acids Research 2012 Apr;40(8):3704-13.
Valdmanis PN, Dupré N, Lachance M, Stochmanski S, Belzil VV, P.A. Dion PA, I. Thiffault, B. Brais, L. Weston, L. Saint-Amant, M.E. Samuels and Rouleau GA. A mutation in the RNF170 gene causes autosomal dominant sensory ataxia. Brain 2011 Feb;134(Pt 2):602-7.
Kwiatkowski TJ Jr, Bosco DA, Leclerc AL, Tamrazian E, Vanderburg CR, Russ C, Davis A, Gilchrist J, Kasarskis EJ, Munsat T, Valdmanis P, Rouleau GA, Hosler BA, Cortelli P, de Jong PJ, Yoshinaga Y, Haines JL, Pericak-Vance MA, Yan J, Ticozzi N, Siddique T, McKenna-Yasek D, Sapp PC, Horvitz HR, Landers JE, Brown RH Jr. Mutations in the FUS gene on chromosome 16 cause familial amyotrophic lateral sclerosis. Science 2009 Feb 27;323(5918):1205-8.
Kabashi E*, Valdmanis PN*, Dion PA, Spiegelman D, McConkey BJ, Vande Velde C, Bouchard J-P, Lacomblez L, Pochigayeva K, Salachas F, Pradat P-F, Camu W, Meininger V, Dupré N and Rouleau GA. TARDBP mutations in sporadic and familial ALS patients. Nature Genetics 2008 May; 40(5):572-4.
Valdmanis PN*, Meijer IA*, Reynolds A, Lei A, MacLeod P, Schlesinger D, Zatz M, Reid E, Dion PA, Drapeau P, and Rouleau GA. Mutations in the KIAA0196 gene at the SPG8 locus cause Hereditary Spastic Paraplegia. American Journal of Human Genetics 2007 Jan; 80(1): 152-61.