Results 61 to 70 of about 2,593 (166)

Transgenic mice expressing tunable levels of DUX4 develop characteristic facioscapulohumeral muscular dystrophy-like pathophysiology ranging in severity

Skeletal Muscle, 2020
Background All types of facioscapulohumeral muscular dystrophy (FSHD) are caused by the aberrant activation of the somatically silent DUX4 gene, the expression of which initiates a cascade of cellular events ultimately leading to FSHD pathophysiology ...
Takako I. Jones, Guo-Liang Chew, Pamela Barraza-Flores, Spencer Schreier, Monique Ramirez, Ryan D. Wuebbles, Dean J. Burkin, Robert K. Bradley, Peter L. Jones   +8 more
doaj   +1 more source

Flavones provide resistance to DUX4-induced toxicity via an mTor-independent mechanism

Cell Death and Disease, 2023
Facioscapulohumeral muscular dystrophy (FSHD) is among the most common of the muscular dystrophies, affecting nearly 1 in 8000 individuals, and is a cause of profound disability.
Justin Cohen, Shushu Huang, Katherine E. Koczwara, Kristen T. Woods, Vincent Ho, Keryn G. Woodman, Jack L. Arbiser, Katelyn Daman, Monkol Lek, Charles P. Emerson, Alec M. DeSimone   +10 more
doaj   +1 more source

Nanopore direct RNA sequencing and the epitranscriptome: Advances in mapping native RNA landscapes

iMeta, Volume 5, Issue 3, June 2026.
Nanopore direct RNA sequencing advances transcriptomics by capturing full‐length transcripts and multiple RNA modifications; this review details its principles, workflows, tools, applications, challenges, and future research potential. Abstract Nanopore direct RNA sequencing (DRS) has transformed transcriptomics by enabling single‐molecule, long‐read ...
Tianyuan Zhang, Jia Li, Chao Tang, You Wu, Hao Wu, Xi‐Tong Zhu, Ziyang Luo, Hang Qin, Lishan Ding, Yu Zeng, Shiou Yih Lee, Xiaotao Shen, Shiwen Gao, Zhaoyang Tian, Qian Tang, Mian Li, Muhammad Tahir Ul Qamar, Yang Dong, Komivi Dossa, Yaxuan Zhang, Hu Chen, Sanqi An, Xiang Yu, Lu Chen, Dingjie Wang, Shengli Li, Ling‐Ling Chen, Yanqiang Li   +27 more
wiley   +1 more source

27‐color flow cytometry for measurable residual disease detection in B‐cell lymphoblastic leukemia

Cytometry Part B: Clinical Cytometry, EarlyView.
Ryan C. Shean, Patricia Davis, Jeffery Jacobsen, David P. Ng   +3 more
wiley   +1 more source

Organoids: From Bench to Bedside Applications

MedComm, Volume 7, Issue 6, June 2026.
Organoids, as a groundbreaking biomedical research platform, utilize adult stem cells (ASCs), embryonic stem cells (ESCs), and induced pluripotent stem cells (iPSCs) as sources. By integrating specific growth and differentiation signals within an extracellular matrix (e.g., Matrigel), organoids guide cells to self‐assemble into three‐dimensional ...
Kelin Li, Rui Cao, Maochen Li, Zichao Tian, Huahao Fan, Bixia Hong, Xiaojuan Liu   +6 more
wiley   +1 more source

Quantitative proteomics reveals key roles for post-transcriptional gene regulation in the molecular pathology of facioscapulohumeral muscular dystrophy

eLife, 2019
DUX4 is a transcription factor whose misexpression in skeletal muscle causes facioscapulohumeral muscular dystrophy (FSHD). DUX4’s transcriptional activity has been extensively characterized, but the DUX4-induced proteome remains undescribed.
Sujatha Jagannathan, Yuko Ogata, Philip R Gafken, Stephen J Tapscott, Robert K Bradley   +4 more
doaj   +1 more source

Venetoclax in Pediatric and Young Adult Patients With Relapsed/Refractory Solid Tumors: Results of a Phase 1 Study

Pediatric Blood &Cancer, Volume 73, Issue 5, May 2026.
ABSTRACT Background B‐cell lymphoma 2 (BCL‐2) is overexpressed in certain solid tumors (including neuroblastoma), representing a promising target. Venetoclax is a first‐in‐class, oral, highly selective BCL‐2 inhibitor. We report safety, pharmacokinetics, and efficacy of venetoclax in children and young adults with relapsed/refractory solid tumors ...
Daniel A. Morgenstern, Giuseppe Barone, Nadege Corradini, Sara M. Federico, Christian Flotho, Nicolas U. Gerber, Seong Lin Khaw, Margaret E. Macy, Andrew E. Place, Kieuhoa T. Vo, Mohamed Badawi, Xin Chen, Adam Luo, Mark A. Rocco, Jeremy A. Ross, Frank Scherer, Gauri Sunkersett, Maika Onishi, Kelly C. Goldsmith   +18 more
wiley   +1 more source

Expression patterns of FSHD-causing DUX4 and myogenic transcription factors PAX3 and PAX7 are spatially distinct in differentiating human stem cell cultures

Skeletal Muscle, 2017
Background Facioscapulohumeral muscular dystrophy (FSHD) is most commonly inherited in an autosomal dominant pattern and caused by the abnormal expression of DUX4 in skeletal muscle.
Premi Haynes, Kelly Kernan, Suk-Lin Zhou, Daniel G. Miller   +3 more
doaj   +1 more source

Homologous Transcription Factors DUX4 and DUX4c Associate with Cytoplasmic Proteins during Muscle Differentiation. [PDF]

PLoS ONE, 2016
Hundreds of double homeobox (DUX) genes map within 3.3-kb repeated elements dispersed in the human genome and encode DNA-binding proteins. Among these, we identified DUX4, a potent transcription factor that causes facioscapulohumeral muscular dystrophy ...
Eugénie Ansseau, Jocelyn O Eidahl, Céline Lancelot, Alexandra Tassin, Christel Matteotti, Cassandre Yip, Jian Liu, Baptiste Leroy, Céline Hubeau, Cécile Gerbaux, Samuel Cloet, Armelle Wauters, Sabrina Zorbo, Pierre Meyer, Isabelle Pirson, Dalila Laoudj-Chenivesse, Ruddy Wattiez, Scott Q Harper, Alexandra Belayew, Frédérique Coppée   +19 more
doaj   +1 more source

Chemotherapy‐driven expression of WNT ligands in bone marrow stromal cells contributes to chemoresistance in acute lymphoblastic leukaemia

British Journal of Haematology, Volume 208, Issue 5, Page 1572-1583, May 2026.
Chemotherapy‐induced WNT ligand secretion by bone marrow (BM) stroma drives acute lymphoblastic leukaemia (ALL) chemoresistance. WNT pathway inhibition disrupts this cross‐talk and restores therapeutic response, highlighting WNT inhibitors as a promising strategy to prevent relapse in ALL.
Foteini Kalampalika, Amanda Jiménez‐Pompa, Raúl Sánchez‐Lanzas, Bela Patel, Miguel Ganuza   +4 more
wiley   +1 more source