Results 71 to 80 of about 3,423 (190)

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

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

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

DUX4-bound motifs are evolutionarily conserved.

, 2013
(A) DUX4 motifs are conserved among placental mammals. We obtained phylogenetic conservation scores for placental mammals (phyloP scores) via the UCSC genome browser for the top-scoring DUX4 motif in each of the 63,795 DUX4 ChIP-seq peaks, along with ...
Zizhen Yao (48910), Janet M. Young (79201), Linda N. Geng (360242), Rabi Tawil (169550), Jennifer L. Whiddon (488099), Silvère M. van der Maarel (1389870), Lauren Snider (360240), Bhavatharini Kasinathan (488100), Judit Balog (262070), Stephen J. Tapscott (269326)   +9 more
core   +1 more source

Transgenic Drosophila for Investigating DUX4 and FRG1, Two Genes Associated with Facioscapulohumeral Muscular Dystrophy (FSHD).

PLoS ONE, 2016
Facioscapulohumeral muscular dystrophy (FSHD) is typically an adult onset dominant myopathy. Epigenetic changes in the chromosome 4q35 region linked to both forms of FSHD lead to a relaxation of repression and increased somatic expression of DUX4-fl ...
Takako I Jones, Megan Parilla, Peter L Jones   +2 more
doaj   +1 more source

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

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

Clinical Characteristics and Prognostic Risk Factors for Pediatric B‐Cell Lymphoblastic Lymphoma: A Multicenter Retrospective Cohort Study for China Net Childhood Lymphoma

Pediatric Blood &Cancer, Volume 73, Issue 4, April 2026.
ABSTRACT Background B‐cell lymphoblastic lymphoma (B‐LBL) represents a rare variety of non‐Hodgkin lymphoma, with limited research on its biology, progression, and management. Methods A retrospective analysis was performed on the clinical characteristics of 256 patients aged ≤18 years who received treatment under the China Net Childhood Lymphoma (CNCL)‐
Zhijuan Liu, Jian Wang, Ling Jin, Yanlong Duan, Fu Li, Yueping Jia, Leping Zhang, Xiaojun Yuan, Wei Liu, Ying Liu, Ansheng Liu, Mincui Zheng, Yunpeng Dai, Lian Jiang, Lirong Sun, Xiaowen Zhai, Rong Liu, Chunju Zhou, Zifen Gao, Yonghong Zhang, Xiaomei Yang   +20 more
wiley   +1 more source

High-throughput screening identifies inhibitors of DUX4-induced myoblast toxicity [PDF]

, 2014
BACKGROUND: Facioscapulohumeral muscular dystrophy (FSHD) is caused by epigenetic alterations at the D4Z4 macrosatellite repeat locus on chromosome 4, resulting in inappropriate expression of the DUX4 protein.
Bosnakovski, Darko, Erik A Toso, Toso, Erik, Darko Bosnakovski, Walters, Michael, Kyba, Michael, Strasser, Jessica, Choi, Si Ho, Jessica M Strasser, Michael A Walters, Si Choi, Michael Kyba   +11 more
core   +1 more source

NuRD and CAF-1-mediated silencing of the D4Z4 array is modulated by DUX4-induced MBD3L proteins

eLife, 2018
The DUX4 transcription factor is encoded by a retrogene embedded in each unit of the D4Z4 macrosatellite repeat. DUX4 is normally expressed in the cleavage-stage embryo, whereas chromatin repression prevents DUX4 expression in most somatic tissues ...
Amy E Campbell, Sean C Shadle, Sujatha Jagannathan, Jong-Won Lim, Rebecca Resnick, Rabi Tawil, Silvère M van der Maarel, Stephen J Tapscott   +7 more
doaj   +1 more source