Results 41 to 50 of about 14,685,807 (300)

Development of therapeutic-grade small interfering RNAs by chemical engineering

open access: yesFrontiers in Genetics, 2012
Recent successes in clinical trials have provided important proof of concept that small interfering RNAs indeed constitute a new promising class of therapeutics.
Jesper Bertram Bramsen, Jørgen eKjems
doaj   +1 more source

Off‐target effects of engineered nucleases [PDF]

open access: yesThe FEBS Journal, 2016
Recent advances in gene editing with engineered nucleases have transformed our ability to manipulate the genome from diverse organisms for applications ranging from biomedical research to disease treatment. A major complication with these engineered nucleases is the binding of the nuclease to unintended genomic sites that share sequence homology with ...
openaire   +2 more sources

Terminal bridging of siRNA duplex at the ribose 2′ position controls strand bias and target sequence preference

open access: yesMolecular Therapy: Nucleic Acids, 2023
Small interfering RNA (siRNA) and short hairpin RNA (shRNA) are widely used as RNA interference (RNAi) reagents. Recently, truncated shRNAs that trigger RNAi in a Dicer-independent manner have been developed.
Atsushi Shibata   +6 more
doaj   +1 more source

Off-target effects of siRNA specific for GFP [PDF]

open access: yesBMC Molecular Biology, 2008
Gene knock down by RNAi is a highly effective approach to silence gene expression in experimental as well as therapeutic settings. However, this widely used methodology entails serious pitfalls, especially concerning specificity of the RNAi molecules.We tested the most widely used control siRNA directed against GFP for off-target effects and found that
Tschuch, Cordula   +8 more
openaire   +2 more sources

Off-target prediction to p.Trp402* sgRNA by predictors and filter criterias.

open access: yes, 2022
Graphic illustration of the off-target prediction to p.Trp402* sgRNA before and after the filtering steps. The image shows the number of sequences returned for each of the five predictors and the number of sequences after the filtering step following the
Ursula Matte (328018)   +2 more
core   +1 more source

Off-Target Effects of Mirabegron on Muscarinic Receptors

open access: yesFuture Pharmacology
Older adults with multiple diseases are likely to be prescribed multiple medications including anticholinergic agents, which are frequently prescribed to manage conditions such as overactive bladder and chronic obstructive pulmonary disease and Parkinson’
Shizuo Yamada   +3 more
doaj   +1 more source

Off-target site analysis.

open access: yes, 2018
(A) The target site (T) and ten predicted off-target sites (OT) for sgRNA91/Cas9. OT1~OT10 represents 10 potential off-target sites, and T represents target site.
Hongming Yuan (2200930)   +20 more
core   +1 more source

Translation of CRISPR Genome Surgery to the Bedside for Retinal Diseases

open access: yesFrontiers in Cell and Developmental Biology, 2018
In recent years, there has been accelerated growth of clustered regularly interspaced short palindromic repeats (CRISPR) genome surgery techniques. Genome surgery holds promise for diseases for which a cure currently does not exist.
Christine L. Xu   +15 more
doaj   +1 more source

On-target and Off-target-based Toxicologic Effects [PDF]

open access: yesToxicologic Pathology, 2012
Adverse toxicologic effects are categorized as chemical-based, on-target, or off-target effects. Chemical-based toxicity is defined as toxicity that is related to the physicochemical characteristics of a compound and its effects on cellular organelles, membranes, and/or metabolic pathways.
openaire   +2 more sources

Off-target sequences

open access: yes, 2014
Supplemental file with off-target sequences obtained using primers designed to specifically amplify bacterial endosymbionts.
Robert W. Bryson (3259062)
core   +1 more source

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