Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS
Iannitti T, Scarrott JM, Likhite S, Coldicott IRP, Lewis KE, Heath PR, Higginbottom A, Myszczynska MA, Milo M, Hautbergue GM, Meyer K, Kaspar BK, Ferraiuolo L, Shaw PJ, Azzouz M. Mol Ther Nucleic Acids. 2018 Sep 7;12:75-88. PubMed
A Novel lncRNA, LINC00460, Affects Cell Proliferation and Apoptosis by Regulating KLF2 and CUL4A Expression in Colorectal Cancer
Lian Y, Yan C, Xu H, Yang J, Yu Y, Zhou J, Shi Y, Ren J, Ji G, Wang K. Mol Ther Nucleic Acids. 2018 Sep 7;12:684-697. PubMed
lncRNA PFAR Promotes Lung Fibroblast Activation and Fibrosis by Targeting miR-138 to Regulate the YAP1-Twist Axis
Zhao X, Sun J, Chen Y, Su W, Shan H, Li Y, Wang Y, Zheng N, Shan H, Liang H. Mol Ther. 2018 Sep 5;26(9):2206-2217. PubMed
MicroRNA-183 and microRNA-96 are associated with autoimmune responses by regulating T cell activation
Thiel J, Alter C, Luppus S, Eckstein A, Tan S, FĂ¼hrer D, Pastille E, Westendorf AM, Buer J, Hansen W. J Autoimmun. 2018 Sep 7. pii: S0896-8411(18)30302-0. PubMed
Topical Lyophilized Targeted Lipid Nanoparticles in the Restoration of Skin Barrier Function following Burn Wound
Li J, Ghatak S, El Masry MS, Das A, Liu Y, Roy S, Lee RJ, Sen CK. Mol Ther. 2018 Sep 5;26(9):2178-2188. PubMed
AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model
Evers MM, Miniarikova J, Juhas S, Vallès A, Bohuslavova B, Juhasova J, Skalnikova HK, Vodicka P, Valekova I, Brouwers C, Blits B, Lubelski J, Kovarova H, Ellederova Z, van Deventer SJ, Petry H, Motlik J, Konstantinova P. Mol Ther. 2018 Sep 5;26(9):2163-2177. PubMed
Ribosomal Protein S3 Gene Silencing Protects Against Cigarette Smoke-Induced Acute Lung Injury
Dong J, Liao W, Peh HY, Tan WSD, Zhou S, Wong WSF. Mol Ther Nucleic Acids. 2018 Sep 7;12:370-380. PubMed
Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
Dulla K, Aguila M, Lane A, Jovanovic K, Parfitt DA, Schulkens I, Chan HL, Schmidt I, Beumer W, Vorthoren L, Collin RWJ, Garanto A, Duijkers L, Brugulat-Panes A, Semo M, Vugler AA, Biasutto P, Adamson P, Cheetham ME. Mol Ther Nucleic Acids. 2018 Sep 7; ...
Morpholino Antisense Oligomers as a Potential Therapeutic Option for the Correction of Alternative Splicing in PMD, SPG2, and HEMS
Tantzer S, Sperle K, Kenaley K, Taube J, Hobson GM. Mol Ther Nucleic Acids. 2018 Sep 7;12:420-432. PubMed
Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification
Vaidyanathan S, Azizian KT, Haque AKMA, Henderson JM, Hendel A, Shore S, Antony JS, Hogrefe RI, Kormann MSD, Porteus MH, McCaffrey AP. Mol Ther Nucleic Acids. 2018 Sep 7;12:530-542. PubMed