Single Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting GalNAc-siRNA Conjugate, Vutrisiran, in Healthy Subjects.
Habtemariam BA, Karsten V, Attarwala H, Goel V, Melch M, Clausen VA, Garg P, Vaishnaw AK, Sweetser MT, Robbie GJ, Vest J. Clin Pharmacol Ther. 2020 Jun 29. Pubmed
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
Miller T, Cudkowicz M, Shaw PJ, Andersen PM, Atassi N, Bucelli RC, Genge A, Glass J, Ladha S, Ludolph AL, Maragakis NJ, McDermott CJ, Pestronk A, Ravits J, Salachas F, Trudell R, Van Damme P, Zinman L, Bennett CF, Lane R, Sandrock A, Runz H, ...
Hybridization Liquid Chromatography-Tandem Mass Spectrometry: An Alternative Bioanalytical Method for Antisense Oligonucleotide Quantitation in Plasma and Tissue Samples.
Li P, Gong Y, Kim J, Liu X, Gilbert J, Kerns HM, Groth R, Rooney M. Anal Chem. 2020 Jul 17. Pubmed
Increased PIP3 activity blocks nanoparticle mRNA delivery.
Paunovska K, Da Silva Sanchez A, Foster MT, Loughrey D, Blanchard EL, Islam FZ, Gan Z, Mantalaris A, Santangelo PJ, Dahlman JE. Sci Adv. 2020 Jul 22;6(30):eaba5672. Pubmed
Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptide.
Nikan M, Tanowitz M, Dwyer CA, Jackson M, Gaus H, Swayze E, Rigo F, Seth PP, Prakash TP. J Med Chem. 2020 Jul 17. Pubmed
Evolution of abiotic cubane chemistries in a nucleic acid aptamer allows selective recognition of a malaria biomarker.
Cheung YW, Röthlisberger P, Mechaly AE, Weber P, Levi-Acobas F, Lo Y, Wong AWC, Kinghorn AB, Haouz A, Savage GP, Hollenstein M, Tanner JA. Proc Natl Acad Sci U S A. 2020 Jul 21;117(29):16790-16798. Pubmed
Aptamers with tunable affinity enable single-molecule tracking and localization of membrane receptors on living cancer cells.
Delcanale P, Porciani D, Pujals S, Jurkevich A, Chetrusca A, Tawiah KD, Burke DH, Albertazzi L. Angew Chem Int Ed Engl. 2020 Jul 6. Pubmed
Discovery and evolution of RNA and XNA reverse transcriptase function and fidelity.
Houlihan G, Arangundy-Franklin S, Porebski BT, Subramanian N, Taylor AI, Holliger P. Nat Chem. 2020 Jul 20. Pubmed
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing.
Mok BY, de Moraes MH, Zeng J, Bosch DE, Kotrys AV, Raguram A, Hsu F, Radey MC, Peterson SB, Mootha VK, Mougous JD, Liu DR. Nature. 2020 Jul 8. doi: 10.1038/s41586-020-2477-4. Pubmed
A dual-deaminase CRISPR base editor enables concurrent adenine and cytosine editing.
GrĂ¼newald J, Zhou R, Lareau CA, Garcia SP, Iyer S, Miller BR, Langner LM, Hsu JY, Aryee MJ, Joung JK. Nat Biotechnol. 2020 Jul;38(7):861-864. Pubmed