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Open AccessJournal ArticleDOI

FDA-Approved Oligonucleotide Therapies in 2017

Cy A. Stein, +1 more
- 03 May 2017 - 
- Vol. 25, Iss: 5, pp 1069-1075
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TLDR
Of all the molecules evaluated as of January 2017, the regulatory authorities assessed that six provided clear clinical benefit in rigorously controlled trials and the story of these six is given in this review.
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This article is published in Molecular Therapy.The article was published on 2017-05-03 and is currently open access. It has received 508 citations till now. The article focuses on the topics: Clinical trial.

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Citations
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Journal ArticleDOI

Block Copolymer Micelles in Nanomedicine Applications.

TL;DR: By shaping these features, polymeric micelles have been propitious for delivering a wide range of therapeutics through effective sensing of targets in the body and adjustment of their properties in response to particular stimuli, modulating the activity of the loaded drugs at the targeted sites, even at the subcellular level.
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Noncoding RNA therapeutics - challenges and potential solutions.

TL;DR: In this article, the authors discuss key challenges facing ncRNA therapeutics, including issues associated with specificity, delivery and tolerability, and focus on promising emerging approaches that aim to boost their success.
Journal ArticleDOI

An overview of active and passive targeting strategies to improve the nanocarriers efficiency to tumour sites.

TL;DR: These properties describing the convenient choice between passive and active targeting mechanisms with details are discussed, illustrated with examples of targeting agents up to preclinical research or clinical advances.
Journal ArticleDOI

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

TL;DR: The chemical modifications and molecular mechanisms that make synthetic nucleic acid drugs possible and lessons learned from recent clinical trials will be summarized.
Journal ArticleDOI

Therapeutic Targeting of Long Non-Coding RNAs in Cancer.

TL;DR: Examples of lncRNAs that demonstrate the diversity of their function in various cancer types are discussed and recent advances in nucleic acid drug development with a focus on oligonucleotide-based therapies as a novel approach to inhibit tumor progression are discussed.
References
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Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase

TL;DR: High-affinity nucleic acid ligands for a protein were isolated by a procedure that depends on alternate cycles of ligand selection from pools of variant sequences and amplification of the bound species.
Journal ArticleDOI

Pegaptanib for Neovascular Age-Related Macular Degeneration

TL;DR: Pegaptanib, an anti-vascular endothelial growth factor therapy, was evaluated in the treatment of neovascular age-related macular degeneration.
Journal ArticleDOI

Fibroblast growth factors, their receptors and signaling.

TL;DR: FGF signaling also appears to play a role in tumor growth and angiogenesis, and autocrine FGF signaling may be particularly important in the progression of steroid hormone-dependent cancers to a hormone-independent state.
Journal ArticleDOI

Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease

TL;DR: Pegaptanib was shown in clinical trials to be effective in treating choroidal neovascularization associated with age-related macular degeneration and has the notable distinction of being the first aptamer therapeutic approved for use in humans, paving the way for future aptamer applications.
Book ChapterDOI

The FGF family of growth factors and oncogenes.

TL;DR: The FGF family includes seven members that share a varying degree of homology at the protein level and appear to have a similar broad mitogenic spectrum and promote the proliferation of a variety of cells of mesodermal and neuroectodermal origin.
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