Nusinersen Initiated in Infants During the Presymptomatic Stage of Spinal Muscular Atrophy
De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, Kirschner J, Kuntz NL, Parsons JA, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Staropoli JF, Kerr D, Sandrock AW, Stebbins C, Petrillo M, Braley ...
Delivery of Macromolecular Therapeutics: Tackling a Billion Year Old Barrier
Presenter:Â Steven Dowdy (UCSD School of Medicine) Date:Â December 17, 2019 Description: All macromolecular therapeutics, including ASOs, siRNAs, peptides, proteins, CRISPR, mRNA and non-viral DNA vectors, are taken up into cells by endocytosis. However, <1% to none of the endocytosed therapeutic cargo escapes into the cytoplasm ...
The changing paradigm of intron retention: regulation, ramifications and recipes.
Monteuuis G, Wong JJL, Bailey CG, Schmitz U, Rasko JEJ. Nucleic Acids Res. 2019 Nov 14. PubMed
Aptamers as Modular Components of Therapeutic Nucleic Acid Nanotechnology.
Panigaj M, Johnson MB, Ke W, McMillan J, Goncharova EA, Chandler M, Afonin KA. ACS Nano. 2019 Nov 26;13(11):12301-12321. PubMed
The Role of Non-coding RNAs in Oncology.
Slack FJ, Chinnaiyan AM. Cell. 2019 Nov 14;179(5):1033-1055. PubMed
The biogenesis, biology and characterization of circular RNAs.
Kristensen LS, Andersen MS, Stagsted LVW, Ebbesen KK, Hansen TB, Kjems J. Nat Rev Genet. 2019 Nov;20(11):675-691. PubMed
Independent control of the thermodynamic and kinetic properties of aptamer switches.
Wilson BD, Hariri AA, Thompson IAP, Eisenstein M, Soh HT. Nat Commun. 2019 Nov 7;10(1):5079. PubMed
Mild Innate Immune Activation Overrides Efficient Nanoparticle-Mediated RNA Delivery.
Lokugamage MP, Gan Z, Zurla C, Levin J, Islam FZ, Kalathoor S, Sato M, Sago CD, Santangelo PJ, Dahlman JE. Adv Mater. 2019 Nov 19:e1904905. PubMed
R-Loops Promote Antisense Transcription across the Mammalian Genome.
Tan-Wong SM, Dhir S, Proudfoot NJ. Mol Cell. 2019 Nov 21;76(4):600-616.e6. PubMed
Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.
González-Duarte A, Berk JL, Quan D, Mauermann ML, Schmidt HH, Polydefkis M, Waddington-Cruz M, Ueda M, Conceição IM, Kristen AV, Coelho T, Cauquil CA, Tard C, Merkel M, Aldinc E, Chen J, Sweetser MT, Wang JJ, Adams D. J Neurol. 2019 Nov 14. PubMed ...
