Results 11 to 20 of about 68,586 (284)

Antisense oligonucleotides as therapeutic agents [PDF]

open access: bronzeJournal of Cellular Physiology, 1999
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 ...
Umberto Galderisi   +2 more
openalex   +6 more sources

Antisense oligonucleotides for neurodegeneration [PDF]

open access: yesScience, 2020
Promising clinical results for Huntington's disease give hope for other ...
Leavitt, BR, Tabrizi, SJ
openaire   +4 more sources

Antisense oligonucleotides [PDF]

open access: yesNeurology Genetics, 2019
There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine.
Eric Vallabh Minikel   +2 more
openaire   +3 more sources

Oligonucleotide Formulations Prepared by High-Speed Electrospinning: Maximizing Loading and Exploring Downstream Processability

open access: yesPharmaceutics, 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

Antisense oligonucleotides in neurological disorders [PDF]

open access: yesTherapeutic Advances in Neurological Disorders, 2018
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
openaire   +3 more sources

Preparation of 5'-O-(1-Thiotriphosphate)-modified oligonucleotides using polymerase-endonuclease amplification reaction (PEAR). [PDF]

open access: yesPLoS 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

Taking control of gene expression with light-activated oligonucleotides

open access: yesBioTechniques, 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

How late is too late for treatment?

open access: yeseLife, 2023
Experiments on mice suggest that an approach called antisense oligonucleotide therapy may be able to treat some symptoms of Angelman syndrome, including problems with epilepsy and sleep.
Lawrence T Reiter
doaj   +1 more source

Identification of sequence motifs significantly associated with antisense activity

open access: yesBMC Bioinformatics, 2007
Background Predicting the suppression activity of antisense oligonucleotide sequences is the main goal of the rational design of nucleic acids. To create an effective predictive model, it is important to know what properties of an oligonucleotide ...
Peek Andrew S, McQuisten Kyle A
doaj   +1 more source

Antisense Therapy for Infectious Diseases

open access: yesCells, 2023
Infectious diseases, particularly Tuberculosis (TB) caused by Mycobacterium tuberculosis, pose a significant global health challenge, with 1.6 million reported deaths in 2021, making it the most fatal disease caused by a single infectious agent. The rise
Lwanda Abonga Buthelezi   +4 more
doaj   +1 more source

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