Hydrocephalus Complicating Intrathecal Antisense Oligonucleotide Therapy for Huntington's Disease
Sign Up to like & getrecommendations! Published in 2020 at "Movement Disorders"
DOI: 10.1002/mds.28359
Abstract: Funding sources and conflict of interest – This trial was funded initially by Ionis and subsequently by Roche. The authors received no additional funding for this work and the authors declare that there are no… read more here.
Keywords: oligonucleotide therapy; complicating intrathecal; antisense oligonucleotide; hydrocephalus complicating ... See more keywords
Development and Clinical Applications of Antisense Oligonucleotide Gapmers.
Sign Up to like & getrecommendations! Published in 2020 at "Methods in molecular biology"
DOI: 10.1007/978-1-0716-0771-8_2
Abstract: DNA-like molecules called antisense oligonucleotides have opened new treatment possibilities for genetic diseases by offering a method of regulating gene expression. Antisense oligonucleotides are often used to suppress the expression of mutated genes which may… read more here.
Keywords: treatment; chemistry; antisense oligonucleotide; antisense oligonucleotides ... See more keywords
Knocking Down Long Noncoding RNAs Using Antisense Oligonucleotide Gapmers.
Sign Up to like & getrecommendations! Published in 2020 at "Methods in molecular biology"
DOI: 10.1007/978-1-0716-0771-8_3
Abstract: Long noncoding RNAs (lncRNAs) are a class of RNA with 200 nucleotides or longer that are not translated into protein. lncRNAs are highly abundant; a study estimates that at least four times more lncRNAs are… read more here.
Keywords: knocking long; long noncoding; using antisense; rnas using ... See more keywords
Antisense Oligonucleotide Targeting of 3'-UTR of mRNA for Expression Knockdown.
Sign Up to like & getrecommendations! Published in 2018 at "Methods in molecular biology"
DOI: 10.1007/978-1-4939-8651-4_6
Abstract: With the recent conditional approval of an antisense oligonucleotide (AON) that restores the reading frame of DMD transcript in a subset of Duchenne muscular dystrophy patients, it has been established that AONs sharing similar chemistry… read more here.
Keywords: targeting utr; utr mrna; oligonucleotide targeting; expression ... See more keywords
Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders
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DOI: 10.1007/s40265-020-01363-3
Abstract: Neuromuscular disorders include a wide range of diseases affecting the peripheral nervous system, which are primarily characterized by progressive muscle weakness and wasting. While there were no effective therapies until recently, several therapeutic approaches have… read more here.
Keywords: status antisense; current status; neuromuscular disorders; antisense oligonucleotide ... See more keywords
Toxicologic evaluation of repetitive 4‐week intravenous injections of midkine antisense oligonucleotide nanoliposomes in rats
Sign Up to like & getrecommendations! Published in 2019 at "Regulatory Toxicology and Pharmacology"
DOI: 10.1016/j.yrtph.2019.01.030
Abstract: Abstract Midkine antisense oligonucleotide (MK‐ASODN) nanoliposomes have previously been shown to have inhibitory activity against hepatocellular carcinoma growth. Herein we report the 4‐week sub‐chronic toxicity of MK‐ASODN nanoliposomes in SD rats. The adverse effects included… read more here.
Keywords: week; asodn nanoliposomes; nanoliposomes rats; midkine antisense ... See more keywords
Activity and Tissue Distribution of Antisense Oligonucleotide CT102 Encapsulated with Cytidinyl/Cationic Lipid against Hepatocellular Carcinoma.
Sign Up to like & getrecommendations! Published in 2022 at "Molecular pharmaceutics"
DOI: 10.1021/acs.molpharmaceut.2c00026
Abstract: Insulin-like growth factor 1 receptor (IGF1R), a cell surface receptor with tyrosine kinase (TK) activity, has ligands abnormally expressed in acute leukemia, multiple myeloma, breast, prostate, cervical, and nonsmall cell lung cancers, Ewing's sarcoma, and… read more here.
Keywords: mix ct102; antisense oligonucleotide; cell; cationic lipid ... See more keywords
Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
Sign Up to like & getrecommendations! Published in 2017 at "Nature Neuroscience"
DOI: 10.1038/nn.4508
Abstract: Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases. read more here.
Keywords: spinal muscular; drug; antisense oligonucleotide; nusinersen antisense ... See more keywords
A new antisense oligonucleotide therapy?
Sign Up to like & getrecommendations! Published in 2018 at "Nature Reviews Rheumatology"
DOI: 10.1038/s41584-018-0128-z
Abstract: from patients with OA compared with undamaged control cartilage. Furthermore, in the destabilization of the medial meniscus mouse model of OA, intra-articular adenoviral delivery of Itgbl1 reduced cartilage damage and osteophyte formation. “As a long-term… read more here.
Keywords: oligonucleotide therapy; antisense oligonucleotide; new antisense;
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect
Sign Up to like & getrecommendations! Published in 2018 at "Nature Medicine"
DOI: 10.1038/s41591-018-0295-0
Abstract: Photoreceptor ciliopathies constitute the most common molecular mechanism of the childhood blindness Leber congenital amaurosis. Ten patients with Leber congenital amaurosis carrying the c.2991+1655A>G allele in the ciliopathy gene centrosomal protein 290 (CEP290) were treated… read more here.
Keywords: leber congenital; antisense oligonucleotide; photoreceptor; congenital amaurosis ... See more keywords
Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis
Sign Up to like & getrecommendations! Published in 2022 at "Nature Medicine"
DOI: 10.1038/s41591-021-01615-z
Abstract: Fused in sarcoma (FUS) is an RNA-binding protein that is genetically and pathologically associated with rare and aggressive forms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). To explore the mechanisms by which mutant… read more here.
Keywords: lateral sclerosis; amyotrophic lateral; antisense oligonucleotide; fus ... See more keywords