Tissue pharmacokinetics of antisense oligonucleotides [PDF]
Pharmacokinetics (PK) of antisense oligonucleotides (ASOs) is characterized by rapid distribution from plasma to tissue and slow terminal plasma elimination driven by re-distribution from tissue. Quantitative understanding of tissue PK and RNA knockdown for various ASO chemistries, conjugations, and administration routes is critical for successful drug
Erica Bäckström+7 more
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erbB-2 antisense oligonucleotides inhibit the proliferation of breast carcinoma cells with erbB-2 oncogene amplification. [PDF]
Amplification and overexpression of the erbB-2 oncogene is an unfavourable prognostic marker in human breast cancer and occurs in approximately 25% of breast carcinomas.
Bacus, S. S.+3 more
core +3 more sources
Accessible light-controlled knockdown of cell-free protein synthesis using phosphorothioate-caged antisense oligonucleotides [PDF]
Light-activated antisense oligonucleotides have been developed to induce gene knockdown in living cells, however, their synthesis remains challenging and application in cell-free systems is underexplored.
Denis Hartmann, Michael J. Booth
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Antisense oligonucleotide inhibition of Heat Shock Protein (HSP) 47 improves bleomycin-induced pulmonary fibrosis in rats [PDF]
Background The most common pathologic form of pulmonary fibrosis arises from excessive deposition of extracellular matrix proteins such as collagen. The 47 kDa heat shock protein 47 (HSP47) is a collagen-specific molecular chaperone that has been shown ...
Noguchi Takayuki+3 more
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Antisense oligonucleotides as therapeutic agents [PDF]
Antisense oligonucleotides can block the expression of specific target genes involved in the development of human diseases. Therapeutic applications of antisense techniques are currently under investigation in many different fields. The use of antisense molecules to modify gene expression is variable in its efficacy and reliability, raising objections ...
GALDERISI, Umberto+2 more
openaire +6 more sources
Antisense oligonucleotides and their applications in rare neurological diseases [PDF]
Rare diseases affect almost 500 million people globally, predominantly impacting children and often leading to significantly impaired quality of life and high treatment costs. While significant contributions have been made to develop effective treatments
Simon McDowall+7 more
doaj +2 more sources
Antisense oligonucleotides in neurological disorders [PDF]
The introduction of genetics revolutionized the field of neurodegenerative and neuromuscular diseases and has provided considerable insight into the underlying pathomechanisms. Nevertheless, effective treatment options have been limited. This changed recently when antisense oligonucleotides (ASOs) could be translated from in vitro and experimental ...
Albert C. Ludolph, Claudia D. Wurster
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Antisense oligonucleotides for neurodegeneration [PDF]
Promising clinical results for Huntington's disease give hope for other ...
Leavitt, BR, Tabrizi, SJ
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Cancer is one of the leading causes of death globally. Epidermal growth factor receptor is one of the proteins involved in cancer cell proliferation, differentiation, and invasion. Antisense oligonucleotides are chemical nucleic acids that bind to target
Akilandeswari Ashwini Balachandran+3 more
doaj +1 more source
Antisense oligonucleotides: recent progress in the treatment of various diseases
Background Antisense oligonucleotides are a promising novel class of therapeutic agents to treat different diseases in living things. They provide an efficient method for making target-selective agents because they change gene expression sequences ...
Chandravadivelu Gopi+2 more
doaj +1 more source