Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

Autor: C. Frank Bennett, John W. Day, Kathie M. Bishop, Eugene Schneider, Frank Rigo, Daniel A. Norris, Richard S. Finkel, Jiri Vajsar, Gene Hung, Jacqueline Montes, Mason Yamashita, Shuting Xia, Darryl C. De Vivo, Claudia A. Chiriboga

Publikováno v: The Lancet. 388:3017-3026

Summary Background Nusinersen is a 2′- O -methoxyethyl phosphorothioate-modified antisense drug being developed to treat spinal muscular atrophy. Nusinersen is specifically designed to alter splicing of SMN2 pre-mRNA and thus increase the amount of

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::252b48c9096ea07b649a3809b33741ff
https://doi.org/10.1016/s0140-6736(16)31408-8

STAT3 antisense oligonucleotide AZD9150 in a subset of patients with heavily pretreated lymphoma: results of a phase 1b trial

Autor: Razelle Kurzrock, Patricia McCoon, Joanna Schmidt, Murali V P Nadella, Paul Lyne, Carl Cook, Tianyuan Zhou, Nathan Fowler, Luis Fayad, Samantha J. Lee, Xiaokun Xiao, Brett P. Monia, Steven G. Hughes, Sarina Anne Piha-Paul, David S. Hong, Anas Younes, John Nemunaitis, Youngsoo Kim, Richard Woessner, Michael A. Curran, Mason Yamashita, Matthew J. Reilley, A. Robert MacLeod, Alexey S. Revenko, Minji Jo, Qinying Liu, Jeff Hsu

Publikováno v: Journal for immunotherapy of cancer, vol 6, iss 1
Journal for Immunotherapy of Cancer
Journal for ImmunoTherapy of Cancer, Vol 6, Iss 1, Pp 1-10 (2018)

Background The Janus kinase (JAK) and signal transduction and activation of transcription (STAT) signaling pathway is an attractive target in multiple cancers. Activation of the JAK-STAT pathway is important in both tumorigenesis and activation of im

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::03714fc6883e9d56aa19fe26e48d996a
https://doi.org/10.1186/s40425-018-0436-5

AZD9150, a next-generation antisense oligonucleotide inhibitor of STAT3 with early evidence of clinical activity in lymphoma and lung cancer

Autor: David S. Hong, Nathan Fowler, Deborah Lawson, Anas Younes, Razelle Kurzrock, Murali V P Nadella, Alexey S. Revenko, Steven G. Hughes, Brett P. Monia, John Nemunaitis, Morvarid Mohseni, Youngsoo Kim, Mason Yamashita, Xiaokun Xiao, A. Robert MacLeod, Minji Jo, David C. Blakey, Joanna Schmidt, Sarina Anne Piha-Paul, Richard Woessner, Tianyuan Zhou, Luis Fayad, Samantha J. Lee, Jeff Hsu, Corinne Reimer

Publikováno v: Science translational medicine. 7(314)

Next-generation sequencing technologies have greatly expanded our understanding of cancer genetics. Antisense technology is an attractive platform with the potential to translate these advances into improved cancer therapeutics, because antisense oli

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2e13772988b21abd682c848c9d95a107
https://pubmed.ncbi.nlm.nih.gov/27868025

Cross-conjugated cyclopentenone prostaglandins synthesis of Δ7-10-chloro-15-deoxy PGA1 ethyl ester

Autor: Mason Yamashita, Jakob Busch-Petersen, Marcus A. Tius

Publikováno v: Tetrahedron Letters. 39:4219-4222

The cationic cyclopentannelation reaction provides an unconventional but highly efficient strategy for the synthesis of unsaturated prostanoids and their analogs.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::d06fb951c6b23e099743ea62b89aebfa
https://doi.org/10.1016/s0040-4039(98)00789-8

Abstract CT239: Clinical and preclinical evidence of an immune modulating role for the STAT3-targeting ASO AZD9150 and potential to enhance clinical responses to anti-PDL1 therapy

Autor: Margaret Veldman-Jones, Paul Lyne, Gene Hung, Rich Woessner, Vivian Jacobs, Kirsten Bell, Michael Collins, Mason Yamashita, Shaun E. Grosskurth, Patricia McCoon, Chris Womack, Michele Johnstone, Rachel DuPont, Robert A. Macleod

Publikováno v: Cancer Research. 75:CT239-CT239

AZD9150 is a therapeutic Generation 2.5 antisense oligonucleotide (ASO) targeting STAT3 that has completed two phase I clinical studies, in patients with HCC and DLBCL, with durable clinical responses seen in both trials. Biomarker studies using pati

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::f1a867d655bcba4648ba2e1341878de2
https://doi.org/10.1158/1538-7445.am2015-ct239