The art of finding the right drug target: emerging methods and strategies
ZC Jia, X Yang, YK Wu, M Li, D Das… - Pharmacological …, 2024 - pharmrev.aspetjournals.org
Drug targets are specific molecules in biological tissues and body fluids that interact with
drugs. Drug target discovery is a key component of drug discovery and is essential for the …
drugs. Drug target discovery is a key component of drug discovery and is essential for the …
Therapeutic targeting of RNA for neurological and neuromuscular disease
JL Bubenik, MM Scotti, MS Swanson - Genes & Development, 2024 - genesdev.cshlp.org
Neurological and neuromuscular diseases resulting from familial, sporadic, or de novo
mutations have devasting personal, familial, and societal impacts. As the initial product of …
mutations have devasting personal, familial, and societal impacts. As the initial product of …
FSHD muscle shows perturbation in fibroadipogenic progenitor cells, mitochondrial function and alternative splicing independently of inflammation
EN Engquist, A Greco, LAB Joosten… - Human Molecular …, 2024 - academic.oup.com
Facioscapulohumeral muscular dystrophy (FSHD) is a prevalent, incurable myopathy. FSHD
is highly heterogeneous, with patients following a variety of clinical trajectories, complicating …
is highly heterogeneous, with patients following a variety of clinical trajectories, complicating …
[HTML][HTML] The transcription factor DUX4 orchestrates translational reprogramming by broadly suppressing translation efficiency and promoting expression of DUX4 …
DC Hamm, EM Paatela, SR Bennett, CJ Wong… - PLoS …, 2023 - journals.plos.org
Translational control is critical for cell fate transitions during development, lineage
specification, and tumorigenesis. Here, we show that the transcription factor double …
specification, and tumorigenesis. Here, we show that the transcription factor double …
Facioscapulohumeral Dystrophy: Molecular Basis and Therapeutic Opportunities
T Arends, DC Hamm… - Cold Spring …, 2024 - cshperspectives.cshlp.org
Facioscapulohumeral dystrophy (FSHD) is caused by misexpression of the early embryonic
transcription factor Double Homeobox Protein 4 (DUX4) in skeletal muscle. DUX4 is …
transcription factor Double Homeobox Protein 4 (DUX4) in skeletal muscle. DUX4 is …
Muscle eosinophilia is a hallmark of chronic disease in facioscapulohumeral muscular dystrophy
AM Nunes, MM Ramirez… - Human Molecular …, 2024 - academic.oup.com
Facioscapulohumeral muscular dystrophy (FSHD) is a progressive myopathy caused by the
aberrant increased expression of the DUX4 retrogene in skeletal muscle cells. The DUX4 …
aberrant increased expression of the DUX4 retrogene in skeletal muscle cells. The DUX4 …
Systemic Pharmacotherapeutic Treatment of the ACTA1-MCM/FLExDUX4 Preclinical Mouse Model of FSHD
N Lu-Nguyen, S Snowden, L Popplewell… - International Journal of …, 2024 - mdpi.com
Aberrant expression of the double homeobox 4 (DUX4) gene in skeletal muscle
predominantly drives the pathogenesis of facioscapulohumeral muscular dystrophy (FSHD) …
predominantly drives the pathogenesis of facioscapulohumeral muscular dystrophy (FSHD) …
[HTML][HTML] Apabetalone, a Clinical-Stage, Selective BET Inhibitor, Opposes DUX4 Target Gene Expression in Primary Human FSHD Muscle Cells
CD Sarsons, D Gilham, LM Tsujikawa, S Wasiak, L Fu… - Biomedicines, 2023 - mdpi.com
Facioscapulohumeral dystrophy (FSHD) is a muscle disease caused by inappropriate
expression of the double homeobox 4 (DUX4) gene in skeletal muscle, and its downstream …
expression of the double homeobox 4 (DUX4) gene in skeletal muscle, and its downstream …
[HTML][HTML] DUX4, the rockstar of embryonic genome activation?
S Nykänen, S Vuoristo - The International Journal of Developmental …, 2024 - ijdb.ehu.eus
During the initial days of development, the embryo gradually shifts from reliance on
maternally provided RNAs and proteins to regulation of its own development. This transition …
maternally provided RNAs and proteins to regulation of its own development. This transition …
[HTML][HTML] The DUX4–HIF1α Axis in Murine and Human Muscle Cells: A Link More Complex Than Expected
TH Nguyen, M Limpens, S Bouhmidi… - International Journal of …, 2024 - mdpi.com
FacioScapuloHumeral muscular Dystrophy (FSHD) is one of the most prevalent inherited
muscle disorders and is linked to the inappropriate expression of the DUX4 transcription …
muscle disorders and is linked to the inappropriate expression of the DUX4 transcription …