Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis
Drug Design, Development and Therapy, 2019 Veena Mathew,1 Annabel K Wang1,2 1Department of Neurology, UCI ALS and Neuromuscular Center, University of California, Irvine, Orange, CA, USA; 2Neurology Section, Tibor Rubin VA Medical Center, Long Beach, CA, USA Abstract: Hereditary transthyretin ...
Mathew V, Wang AK
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Antisense oligonucleotides targeting Notch2 ameliorate the osteopenic phenotype in a mouse model of Hajdu-Cheney syndrome [PDF]
, 2020 Ernesto Canalis +4 more
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British Journal of Clinical Pharmacology, EarlyView.Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić +4 more
wiley +1 more source
BMEMat, EarlyView.Illustration of 5% S‐vacancy Bi2S3 mediated phosphodiester bonds cleavage in RNA of hepatocellular carcinoma cells, which suppressing ERI3 expression, inhibiting cell proliferation and promoting apoptosis. Abstract Genome‐wide hypertranscription is a hallmark of malignant progression.
Chuncheng Yang +12 more
wiley +1 more source
Recent Insights Into Alzheimer's Disease: Advances in Pathophysiology and Therapeutic Strategies
Brain Health, EarlyView.ABSTRACT Alzheimer's disease (AD) is a complex neurodegenerative disorder driven by interplay among amyloid‐β (Aβ) plaques, tau neurofibrillary tangles, inflammation and comorbid pathologies. Recent advances in biomarkers and therapeutic approvals have reshaped our understanding of its pathophysiology and treatment landscape.
Jiong Shi +3 more
wiley +1 more source
Evolved DNAzymes and Stable Activation Chemistry Enable High‐Efficiency DNA Ligation
Chemistry – A European Journal, EarlyView.In vitro selection of pre‐structured DNA libraries and chemical screening of alternative activation groups yielded trans‐acting DNAzymes with faster ligation rates and enhanced DNA substrate stability. The combination of C3S1 DNAzymes and phosphobenzimidazole‐activated DNA substrates provides improved DNA ligation performance, offering promise for ...
Connor Nurmi +3 more
wiley +1 more source
Antisense oligonucleotide targeting Bcl-2 messenger RNA in cancer: bad drug, bad target, neither or both? [PDF]
, 2009 Yohann Loriot +2 more
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Mini‐Hairpin DNA: Extraordinarily Stable Structure and Its Applications
Chemistry – A European Journal, EarlyView.Mini‐hairpin DNA fragments containing GCGAAGC and GCGAAAGC form exceptionally stable hairpin‐like structures, with only two G−C pairs and one sheared G−A pair, remaining undenatured in 7 M urea and resisting nuclease digestion. These mini‐hairpin sequences frequently appear in replication and transcription origins, influence DNA sequencing fidelity ...
Michiko Kimoto, Ichiro Hirao
wiley +1 more source
STAT3 Antisense Oligonucleotide Remodels the Suppressive Tumor Microenvironment to Enhance Immune Activation in Combination with Anti–PD-L1 [PDF]
, 2020 Theresa A. Proia +22 more
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Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: study protocol for a phase I/II clinical trial [PDF]
, 2023 Takami Ishizuka +11 more
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