Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Neurology. 2020 Mar 5. PubMed
A CRISPR-Cas9-based reporter system for single-cell detection of extracellular vesicle-mediated functional transfer of RNA
de Jong OG, Murphy DE, Mäger I, Willms E, Garcia-Guerra A, Gitz-Francois JJ, Lefferts J, Gupta D, Steenbeek SC, van Rheenen J, El Andaloussi S, Schiffelers RM, Wood MJA, Vader P. Nat Commun. 2020 Feb 28;11(1):1113. PubMed
Interview with Mirjana Kujacic, PhD
Science: Q: Paul Zamecnik and Mary Stephenson in 1978 reported the very first studies on antisense mechanisms of gene silencing. Since the inception of this field – how far have we come – both in basic and therapeutic research & development? A: The original antisense mechanism has been joined
A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis
Rodriguez CM, Wright SE, Kearse MG, Haenfler JM, Flores BN, Liu Y, Ifrim MF, Glineburg MR, Krans A, Jafar-Nejad P, Sutton MA, Bassell GJ, Parent JM, Rigo F, Barmada SJ, Todd PK. Nat Neurosci. 2020 Mar;23(3):386-397. Joint First Authors Prof. Peter K. ...
Coronavirus Highlights Urgent Need for Science-Backed Research
News Release from Mary Ann Liebert, Inc. publishers Coronavirus Highlights Urgent Need for Science-Backed Research As an academic publisher, we publish many important resources relevant to COVID-19 such as Health Security, Viral Immunology, Telemedicine and e-Health, and Vector Borne and Zoonotic Diseases. We are ...
Targeting the IL-17 Receptor Using Liposomal Spherical Nucleic Acids as Topical Therapy for Psoriasis
Liu H, Kang RS, Bagnowski K, Yu JM, Radecki S, Daniel WL, Anderson BR, Nallagatla S, Schook A, Agarwal R, Giljohann DA, Paller AS. J Invest Dermatol. 2020 Feb;140(2):435-444.e4. PubMed
A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis
Rodriguez CM, Wright SE, Kearse MG, Haenfler JM, Flores BN, Liu Y, Ifrim MF, Glineburg MR, Krans A, Jafar-Nejad P, Sutton MA, Bassell GJ, Parent JM, Rigo F, Barmada SJ, Todd PK. Nat Neurosci. 2020 Feb 17. PubMed
A unified allosteric/torpedo mechanism for transcriptional termination on human protein-coding genes
Eaton JD, Francis L, Davidson L, West S. Genes Dev. 2020 Jan 1;34(1-2):132-145. PubMed
Endocytosis Controls siRNA Efficiency: Implications for siRNA Delivery Vehicle Design and Cell-Specific Targeting
Vocelle D, Chan C, Walton SP. Nucleic Acid Ther. 2020 Feb;30(1):22-32. PubMed
Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold
Xie J, Tai PWL, Brown A, Gong S, Zhu S, Wang Y, Li C, Colpan C, Su Q, He R, Ma H, Li J, Ye H, Ko J, Zamore PD, Gao G. Mol Ther. 2020 Feb 5;28(2):422-430. PubMed
