Results 111 to 120 of about 102,021 (242)

POCALI: Prediction and Insight on CAncer LncRNAs by Integrating Multi‐Omics Data with Machine Learning

Small Methods, EarlyView.
Long non‐coding RNAs (lncRNAs) are receiving increasing attention as biomarkers for cancer diagnosis and therapy, highlighting the urgent need for computational methods to accelerate their comprehensive discovery. Here, to better predict and provide functional insight into cancer lncRNAs, a novel interpretable machine‐learning method (POCALI) is ...
Ziyan Rao, Chenyang Wu, Yunxi Liao, Chuan Ye, Shaodong Huang, Dongyu Zhao   +5 more
wiley   +1 more source

An antisense oligonucleotide-based strategy to ameliorate cognitive dysfunction in the 22q11.2 Deletion Syndrome. [PDF]

Elife
Thakur P, Lackinger M, Diamantopoulou A, Rao S, Chen Y, Khalizova K, Ferng A, Mazur C, Kordasiewicz H, Shprintzen RJ, Markx S, Xu B, Gogos JA.   +12 more
europepmc   +1 more source

Patient‐Reported Outcomes in the Phase III OASIS‐HAE Study of Donidalorsen for Hereditary Angioedema

Allergy, EarlyView.
We report an overview of patients' self‐reported quality of life, disease control, and functioning in daily life while taking donidalorsen for HAE in the 24‐week phase 3 OASIS‐HAE trial. Most patients reported clinically meaningful improvements in quality of life and disease control.
Marc A. Riedl, Aaron Yarlas, Laura Bordone, Sabrina Treadwell, Sophie Wang, Kenneth B. Newman, Danny M. Cohn   +6 more
wiley   +1 more source

Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment. [PDF]

Beilstein J Nanotechnol
Alvarez AC, Maguire D, Brannigan RP.
europepmc   +1 more source

Discovery of natural product derivative triptolidiol as a direct NLRP3 inhibitor by reducing K63‐specific ubiquitination

British Journal of Pharmacology, EarlyView.
▪ Background and Purpose NLRP3 is up‐regulated in inflammatory and autoimmune diseases. The development of NLRP3 inhibitors is challenged by the identification of compounds with distinct mechanisms of action avoiding side effects and toxicity. Triptolide is a natural product with multiple anti‐inflammatory activities, but a narrow therapeutic window ...
Mo‐Yu Ding, Chengqing Ning, Shao‐Ru Chen, Hao‐Ran Yin, Jing Xu, Ying Wang   +5 more
wiley   +1 more source

Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments. [PDF]

Am J Hum Genet
Cheerie D, Meserve MM, Beijer D, Kaiwar C, Newton L, Taylor Tavares AL, Verran AS, Sherrill E, Leonard S, Sanders SJ, Blake E, Elkhateeb N, Gandhi A, Liang NSY, Morgan JT, Verwillow A, Verheijen J, Giles A, Williams S, Chopra M, Croft L, Dafsari HS, Davidson AE, Friedman J, Gregor A, Haque B, Lechner R, Montgomery KA, Ryten M, Schober E, Siegel G, Sullivan PJ, Whittle EF, Zardetto B, Yu TW, Synofzik M, Aartsma-Rus A, Costain G, Lauffer MC, N=1 Collaborative.   +39 more
europepmc   +1 more source

Molecular genetics of development studied in the transgenic mouse. [PDF]

, 1989
Gruss, P., Westphal, H.
core   +2 more sources

Site‐specific m6A‐miR‐494‐3p, not unmethylated miR‐494‐3p, compromises blood brain barrier by targeting tight junction protein 1 in intracranial atherosclerosis

British Journal of Pharmacology, EarlyView.
Background and Purpose Intracranial atherosclerosis is one of the most common causes of ischaemic stroke. However, there is a substantial knowledge gap on the development of intracranial atherosclerosis. Intracranial arteries are characterized by an upregulation of tight junctions between endothelial cells, which control endothelial permeability.
Tamar Woudenberg, M. Leontien van der Bent, Veerle Kremer, Ingeborg S. E. Waas, Mat J. A. P. Daemen, Reinier A. Boon, Paul H. A. Quax, A. Yaël Nossent   +7 more
wiley   +1 more source

Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis. [PDF]

Ann Clin Transl Neurol
Ito D, Okada K.
europepmc   +1 more source

Advancing antihypertensive drug development

British Journal of Pharmacology, EarlyView.
Hypertension affects more than a billion individuals worldwide and remains a major cause of cardiovascular morbidity and mortality. Despite advances in therapies, optimal blood pressure control remains elusive for many patients because of treatment resistance, adverse effects, and adherence challenges.
Fay Pu, Yanrong Liu, Fozia Zahir Ahmed, Xin Wang, Darren Green, Hongyuan Zhang   +5 more
wiley   +1 more source