Results 11 to 20 of about 171,175 (241)
Advances in antisense oligonucleotide development for target identification, validation, and as novel therapeutics [PDF]
, 2008 Antisense oligonucleotides (As-ODNs) are single stranded, synthetically prepared strands of deoxynucleotide sequences, usually 18–21 nucleotides in length, complementary to the mRNA sequence of the target gene.
Alirio J. Melendez +3 more
core +8 more sources
Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects [PDF]
Molecular Therapy: Nucleic AcidsNicotinamide N-methyltransferase (NNMT) is one of the methyltransferase family genes. It consumes S-adenosyl-l-methionine (SAM), which is required for DNA methylation and histone methylation for epigenetic regulation, to produce 1-methylnicotinamide from
Tomoaki Hara +13 more
doaj +3 more sources
Pharmacokinetics of antisense oligonucleotide drugs [PDF]
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
Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review
European Journal of Cell Biology, 2023 Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness.
Zorica Nakevska, Toshifumi Yokota
doaj +1 more source
Pharmaceutics, 2023 The aim of this study was to develop antisense oligonucleotide tablet formulations using high-speed electrospinning. Hydroxypropyl-beta-cyclodextrin (HPβCD) was used as a stabilizer and as an electrospinning matrix. In order to optimize the morphology of
Edit Hirsch +13 more
doaj +1 more source
Molecular Therapy: Nucleic Acids, 2018 Duchenne muscular dystrophy (DMD), the most common lethal heritable childhood disease, is caused by mutations in the DMD gene that result in the absence of functional dystrophin protein.
Naoki Watanabe +8 more
doaj +1 more source
Antisense oligonucleotides or oligonucleotide analogues [PDF]
, 2013 The present invention relates to antisense oligonucleotides or oligonucleotide analogues adapted to target pre-mRNA which encodes GNB3s and prevent aberrant splicing events.
Lester, Douglas H.
core +8 more sources
Preparation of 5'-O-(1-Thiotriphosphate)-modified oligonucleotides using polymerase-endonuclease amplification reaction (PEAR). [PDF]
PLoS ONE, 2013 Antisense oligonucleotides (ASODNs) have been widely used as an important tool for regulating gene expression, and developed into therapeutics. Natural ODNs are susceptible to nuclease degradation, nucleic acid analogues, however, have less side effects,
Biao Li +7 more
doaj +1 more source
Molecular Therapy: Nucleic Acids, 2019 Antisense oligonucleotide therapy has been reported to be associated with renal injury. Here, the mechanism of reversible proteinuria was investigated by combining clinical, pre-clinical, and in vitro data.
Manoe J. Janssen +10 more
doaj +1 more source
Taking control of gene expression with light-activated oligonucleotides
BioTechniques, 2007 The recent development of caged oligonucleotides that are efficiently activated by ultraviolet (UV) light creates opportunities for regulating gene expression with very high spatial and temporal resolution.
Ivan J. Dmochowski, XinJing Tang
doaj +1 more source