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, Führer 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
In vivo CRISPR editing with no detectable genome-wide off-target mutations
Akcakaya P, Bobbin ML, Guo JA, Malagon-Lopez J, Clement K, Garcia SP, Fellows MD, Porritt MJ, Firth MA, Carreras A, Baccega T, Seeliger F, Bjursell M, Tsai SQ, Nguyen NT, Nitsch R, Mayr LM, Pinello L, Bohlooly-Y M, Aryee MJ, Maresca M, Joung JK. Nature. ...
Structural Basis for the RNA-Guided Ribonuclease Activity of CRISPR-Cas13d
Zhang C, Konermann S, Brideau NJ, Lotfy P, Wu X, Novick SJ, Strutzenberg T, Griffin PR, Hsu PD, Lyumkis D. Cell. 2018 Sep 20;175(1):212-223.e17. PubMed
A Synthetic Cross-Species CD200R1 Agonist Suppresses Inflammatory Immune Responses In Vivo
Prodeus A, Sparkes A, Fischer NW, Cydzik M, Huang E, Khatri I, Young A, Woo L, Chow CW, Gorczynski R, Gariépy J. Mol Ther Nucleic Acids. 2018 Sep 7;12:350-358. PubMed
