Results 1 to 10 of about 109,114 (209)
Antisense Drugs Make Sense for Neurological Diseases. [PDF]
Annu Rev Pharmacol Toxicol, 2021 The genetic basis for most inherited neurodegenerative diseases has been identified, yet there are limited disease-modifying therapies for these patients. A new class of drugs—antisense oligonucleotides (ASOs)—show promise as a therapeutic platform for treating neurological diseases.
Bennett CF +2 more
europepmc +4 more sources
Development of Antisense Drugs for Dyslipidemia. [PDF]
J Atheroscler Thromb, 2016 Abnormal elevation of low-density lipoprotein (LDL) and triglyceride-rich lipoproteins in plasma as well as dysfunction of anti-atherogenic high-density lipoprotein (HDL) have both been recognized as essential components of the pathogenesis of atherosclerosis and are classified as dyslipidemia.
Yamamoto T, Wada F, Harada-Shiba M.
europepmc +4 more sources
Pharmacokinetics of antisense oligonucleotide drugs
Фармакокинетика и Фармакодинамика, 2013 This review covers the pharmacokinetic characteristics of the various antisense oligonucleotide drugs. A comparison of the pharmacokinetics of drugs first and second generation.
M. R. Khaitov, V. V. Smirnov
doaj +1 more source
AIMS Neuroscience, 2021 Lesch-Nyhan disease (LND) is a rare X-linked inherited neurogenetic disorders of purine metabolic in which the cytoplasmic enzyme, hypoxanthine-guanine phosphoribosyltransferase (HGprt) is defective.
Khue Vu Nguyen
doaj +1 more source
AIMS Neuroscience, 2022 A heterozygous Arg393His point mutation at the reactive site of antithrombin (AT) gene causing thrombosis in a Vietnamese patient is reported and named as Arg393His in AT-Hanoi.
Khue Vu Nguyen
doaj +1 more source
Antisense Oligonucleotides: Concepts and Pharmaceutical Applications
Borneo Journal of Pharmacy, 2023 Antisense oligonucleotides are drugs whose mechanism is based on binding to RNA target sequences. For this purpose, they modify the protein expression through steric hindrance and exon omission.
Ariana Araya +5 more
doaj +1 more source
Natural antisense transcripts as drug targets
Frontiers in Molecular Biosciences, 2022 The recent discovery of vast non-coding RNA-based regulatory networks that can be easily modulated by nucleic acid-based drugs has opened numerous new therapeutic possibilities. Long non-coding RNA, and natural antisense transcripts (NATs) in particular, play a significant role in networks that involve a wide variety of disease-relevant biological ...
Olga Khorkova +7 more
openaire +3 more sources
Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
罕见病研究, 2022 In recent years, antisense oligonucleotide (ASO) has been very active in the field of rare disease research and development, especially in Duchenne muscular dystrophy, where it made a major breakthrough.
XU Tingting +6 more
doaj +1 more source
Antisense PNA accumulates in Escherichia coli and mediates a long post-antibiotic effect [PDF]
, 2007 Antisense agents that target growth-essential genes display surprisingly potent bactericidal properties. In particular, peptide nucleic acid (PNA) and phosphorodiamidate morpholino oligomers linked to cationic carrier peptides are effective in time kill ...
Behmanesh, Mehrdad +9 more
core +2 more sources
β-Amyloid precursor protein (APP) and the human diseases
AIMS Neuroscience, 2019 Several pathophysiological functions of the human β-amyloid precursor protein (APP) have been recently proposed in different human diseases such as neurodevelopmental and neurodegenerative disorders including rare diseases such as autism, fragile X ...
Khue Vu Nguyen
doaj +1 more source