Aptamers: Cutting edge of cancer therapies.

Autor:	Shigdar S; School of Medicine and Centre for Molecular and Medical Research, Deakin University, 75 Pigdons Road, Waurn Ponds, VIC 3216, Australia., Schrand B; TCR(2) Therapeutics, Inc., 100 Binney Street, Cambridge, MA 02142, USA., Giangrande PH; Internal Medicine, University of Iowa, Iowa City, IA 52242, USA; VP Platform Discovery Sciences, Biology, Wave Life Sciences, Cambridge, MA 02138, USA., de Franciscis V; Institute of Genetic and Biomedical Research (IRGB), National Research Council (CNR), Milan, Italy; Initiative for RNA Medicine, Harvard Medical School, Boston, MA 02115, USA. Electronic address: vittorio.defranciscis@irgb.cnr.it.
Jazyk:	angličtina
Zdroj:	Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2021 Aug 04; Vol. 29 (8), pp. 2396-2411. Date of Electronic Publication: 2021 Jun 17.
DOI:	10.1016/j.ymthe.2021.06.010
Abstrakt:	The development of an aptamer-based therapeutic has rapidly progressed following the first two reports in the 1990s, underscoring the advantages of aptamer drugs associated with their unique binding properties. In 2004, the US Food and Drug Administration (FDA) approved the first therapeutic aptamer for the treatment of neovascular age-related macular degeneration, Macugen developed by NeXstar. Since then, eleven aptamers have successfully entered clinical trials for various therapeutic indications. Despite some of the pre-clinical and clinical successes of aptamers as therapeutics, no aptamer has been approved by the FDA for the treatment of cancer. This review highlights the most recent and cutting-edge approaches in the development of aptamers for the treatment of cancer types most refractory to conventional therapies. Herein, we will review (1) the development of aptamers to enhance anti-cancer immunity and as delivery tools for inducing the expression of immunogenic neoantigens; (2) the development of the most promising therapeutic aptamers designed to target the hard-to-treat cancers such as brain tumors; and (3) the development of "carrier" aptamers able to target and penetrate tumors and metastasis, delivering RNA therapeutics to the cytosol and nucleus. Competing Interests: Declaration of interests B.S. is co-founder of Sebastian Bio, which has intellectual property (IP) on the AS1411-siTAP chimera. P.H.G. is employed by Wave Life Sciences and receives salary and shares from Wave Life Sciences. (Copyright © 2021 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.)
Databáze:	MEDLINE
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