TGF‐β inhibition is emerging as a promising cancer therapy, yet translating laboratory success to bedside implementation has suffered significant setbacks. The associated challenges include adverse drug reactions, inadequate predictive models, and activation of alternative signaling pathways.
Faizah A. Alabi+5 more
wiley +1 more source
Recent Progress of Antisense Oligonucleotide Therapy for Superoxide-Dismutase-1-Mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen. [PDF]
Moriyama H, Yokota T.
europepmc +1 more source
Serum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen‐Treated ALS
ABSTRACT Introduction/Aims Cardiac troponin T (cTnT) levels are elevated in the majority of persons with amyotrophic lateral sclerosis (ALS) and increase over time. Neurofilament light chain (NfL) is an established therapy response biomarker in ALS as superoxide dismutase1 (SOD1)‐ALS patients treated with the antisense oligonucleotide tofersen show a ...
Sarah Bernsen+7 more
wiley +1 more source
ABSTRACT Introduction/Aims The biological changes in motor neurons and motor axons that correlate with the clinical benefits of nusinersen, an antisense oligonucleotide, in spinal muscular atrophy (SMA) remain poorly understood. This study aimed to investigate changes in axonal excitability and motor unit number estimation (MUNE) parameters following a
Abir Alaamel+4 more
wiley +1 more source
Bioanalysis of free antisense oligonucleotide payload from antibody-oligonucleotide conjugate by hybridization LC-MS/MS. [PDF]
Jiang D, Li P, Yuan L.
europepmc +1 more source
Functional Differences Between SIRPα Splice Isoforms
SIRPα is expressed in phagocytic cells, and when it recognizes CD47, “don't eat me” signal, it suppresses phagocytosis. We show here that short SIRPα, a less‐studied splice isoform, has a different function from a well‐studied long SIRPα. Short SIRPα may act as a “don't eat me” signal regulator.
Mihoko Kajita+10 more
wiley +1 more source
Fifteen Years of PNPLA3: Transforming Hepatology Through Human Genetics
ABSTRACT Steatotic liver disease (SLD), caused by excess lipid accumulation in hepatocytes, is now a leading global liver condition triggered by metabolic dysfunction, alcohol, toxins and heritable factors. The main genetic determinant is the common PNPLA3 p.I148M variant, which explains a substantial portion of SLD interindividual and interethnic ...
Stefano Romeo, Luca Valenti
wiley +1 more source
Molecular Profiling of Mouse Models of Loss or Gain of Function of the KCNT1 (Slack) Potassium Channel and Antisense Oligonucleotide Treatment. [PDF]
Sun F+12 more
europepmc +1 more source
Alternative Splicing Regulation in Metabolic Disorders
ABSTRACT Alternative splicing (AS) is a fundamental mechanism for enhancing transcriptome diversity and regulating gene expression, crucial for various cellular processes and the development of complex traits. This review examines the role of AS in metabolic disorders, including obesity, weight loss, dyslipidemias, and metabolic syndrome.
Dorota Kaminska
wiley +1 more source
Liver-targeted Angptl4 silencing by antisense oligonucleotide treatment attenuates hyperlipidaemia and atherosclerosis development in APOE*3-Leiden.CETP mice. [PDF]
Modder M+13 more
europepmc +1 more source