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Nanotechnology Could Revolutionize Therapy for Age-Related Macular Degeneration
Published in Bhupinder Singh, Om Prakash Katare, Eliana B. Souto, NanoAgroceuticals & NanoPhytoChemicals, 2018
Afrah Jalil Abd, Shahdeep Kaur, Ripandeep Kaur, Rupinder Kaur Kanwar, Bhupinder Singh, Jagat Rakesh Kanwar
Treatment for AMD requires intraocular administration. Despite the achieved improvement for AMD therapy using the intravitreal approach, the issue for rapid clearance is still undesirable since it requires repeated injection and subsequent risk of surgery, as well as the probability of further requirement of doses to maintain drug release. In order to overcome this obstacle, sustained intraocular drug release has been developed using nanoparticles (Birch and Liang, 2007; Farjo and Ma, 2010; Elsaid et al., 2016). These microscopically sized spheres are made from artificial or natural polymers, as portrayed in Figure 12.3b. The aptamer Pegaptanib (Figure 12.3c) was approved by the US Food and Drug Administration (FDA) to treat AMD, as it has the ability to selectively bind with VEGF and inhibit vascular leakage. Although both the drugs showed some efficacy in slowing disease progression and improving vision, it could further reduce the frequency of intravitreal injection. Nanoparticles loaded with ganciclovir along with nanoparticle-mediated gene delivery were also administered into the vitreous chamber and have been reported to have a prolonged presence in the eye without toxic effects (Kanwar et al., 2014).
Aptamers as Therapeutic Tools in Neurological Diseases
Published in Rakesh N. Veedu, Aptamers, 2017
Lukas Aaldering, Shilpa Krishnan, Sue Fletcher, Stephen D. Wilton, Rakesh N. Veedu
Nucleic acid aptamers have attracted considerable attention in recent years as tools for targeted molecular recognition. These synthetic nucleic acid ligands can fold into three-dimensional shapes (Fig. 6.1) for binding defined molecular targets with specific high-affinity recognition. With potential targets ranging from small chemicals to large proteins, these characteristics make aptamers an attractive platform for applications relating to drug delivery, biosensing, and theranostic. Aptamers are generally selected using systematic evolution of ligands by exponential enrichment (SELEX) [1] procedures. Ever since their introduction to the scientific community, aptamers have been applied in various research fields toward developing novel therapeutics or as tools for diagnostics and imaging [2, 3]. In 2004, Pegaptanib sodium (Macugen) was approved by the FDA for the treatment of age-related macular degeneration (AMD). Due to their functional similarities to antibodies, aptamers are often termed as “chemical antibodies.” However, aptamers possess certain advantages over antibodies, such as stability, production scale, method and time, chemical fabrication, and costs. Furthermore, they are not immunogenic and can be synthesized in vitro in large quantities, up to kilograms. Herein, we describe recent advances in aptamer technology in the field of neurological diseases.
Bio-Inspired DNA Nanoswitches and Nanomachines: Applications in Biosensing and Drug Delivery
Published in Klaus D. Sattler, st Century Nanoscience – A Handbook, 2020
Arnaud Desrosiers, Alexis Vallée-Bélisle
Some of these chemical modifications have already been approved by the FDA. For example, the phosphorothioate linkage is present in Nusinersen, an antisense oligonucleotides drug [137] used to treat spinal muscular atrophy [138]. Pegaptanib is an example of a 2′ modified oligonucleotide that is now used as a therapeutic agent to treat macular degeneration [139]. Another backbone modification, L-DNA (i.e. the mirror image of the natural form D-DNA) is also resistant to nuclease degradation and was successfully used to build DNA nanothermometers, which were used inside living cells [46].
Aptamer based tools for environmental and therapeutic monitoring: A review of developments, applications, future perspectives
Published in Critical Reviews in Environmental Science and Technology, 2020
Błażej Kudłak, Monika Wieczerzak
In 2004, the FDA (Food and Drug Administration) approved Macugen® (pegaptanib), an aptamer based drug used to treat adults who suffer from wet-form age-related macular degeneration. In this treatment, a modified pegylated oligonucleotide binds selectively and strongly to the extracellular form of endothelial growth factor (VEGF165) to inhibit its action. In January 2017, the FDA approved oligonucleotide therapies following assessment that aptamer-based drugs provided a clear benefit in rigorously controlled clinical trials (Stein & Castanotto, 2017).