Antisense Inhibition of Glucagon Receptor by IONIS-GCGRRx Improves Type 2 Diabetes Without Increase in Hepatic Glycogen Content in Patients With Type 2 Diabetes on Stable Metformin Therapy
Morgan ES, Tai LJ, Pham NC, Overman JK, Watts LM, Smith A, Jung SW, Gajdošík M, Krššák M, Krebs M, Geary RS, Baker BF, Bhanot S. Diabetes Care. 2019 Feb 14. pii: dc181343. PubMed
Intradermal delivery of modified mRNA encoding VEGF-A in patients with type 2 diabetes
Gan LM, Lagerström-Fermér M, Carlsson LG, Arfvidsson C, Egnell AC, Rudvik A, Kjaer M, Collén A, Thompson JD, Joyal J, Chialda L, Koernicke T, Fuhr R, Chien KR, Fritsche-Danielson R. Nat Commun. 2019 Feb 20;10(1):871. PubMed
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis
Solomon SD, Adams D, Kristen A, Grogan M, González-Duarte A, Maurer MS, Merlini G, Damy T, Slama MS, Brannagan TH 3rd, Dispenzieri A, Berk JL, Shah AM, Garg P, Vaishnaw A, Karsten V, Chen J, Gollob J, Vest J, Suhr O. Circulation. 2019 Jan 22;139(4):431-443. ...
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect
Cideciyan AV, Jacobson SG, Drack AV, Ho AC, Charng J, Garafalo AV, Roman AJ, Sumaroka A, Han IC, Hochstedler MD, Pfeifer WL, Sohn EH, Taiel M, Schwartz MR, Biasutto P, Wit W, Cheetham ME, Adamson P, Rodman DM, Platenburg G, Tome MD, Balikova I, Nerinckx ...
Integrated Assessment of the Clinical Performance of GalNAc3-Conjugated 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer Experience
Crooke ST, Baker BF, Xia S, Yu RZ, Viney NJ, Wang Y, Tsimikas S, Geary RS. Nucleic Acid Ther. 2018 Dec 20. PubMed
A microRNA-29 mimic (remlarsen) represses extracellular matrix expression and fibroplasia in the skin
Gallant-Behm CL, Piper J, Lynch JM, Seto AG, Hong SJ, Mustoe TA, Maari C, Pestano LA, Dalby CM, Jackson AL, Rubin P, Marshall WS. J Invest Dermatol. 2018 Nov 22. PubMed
STAT3 antisense oligonucleotide AZD9150 in a subset of patients with heavily pretreated lymphoma: results of a phase 1b trial
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Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients
Turner AM, Stolk J, Bals R, Lickliter JD, Hamilton J, Christianson DR, Given BD, Burdon JG, Loomba R, Stoller JK, Teckman JH. J Hepatol. 2018 Aug;69(2):378-384. PubMed
SD-101 in Combination with Pembrolizumab in Advanced Melanoma: Results of a Phase 1b, Multicenter Study
Ribas A, Medina T, Kummar S, Amin A, Kalbasi A, Drabick JJ, Barve M, Daniels GA, Wong DJ, Schmidt EV, Candia AF, Coffman RL, Leung ACF, Janssen RS. Cancer Discov. 2018 Aug 28. PubMed
In Situ Vaccination with a TLR 9 Agonist and Local Low Dose Radiation Induces Systemic Responses in Untreated Indolent Lymphoma
Frank MJ, Reagan PM, Bartlett NL, Gordon LI, Friedberg JW, Czerwinski DK, Long SR, Hoppe RT, Janssen RS, Candia AF, Coffman RL, Levy R. Cancer Discov. 2018 Aug 28. PubMed
