Aptamers: Cutting edge of cancer therapies

被引：88

作者：

Shigdar, Sarah ^{[1
,2
]}

Schrand, Brett ^{[3
]}

Giangrande, Paloma H. ^{[4
,5
]}

de Franciscis, Vittorio ^{[6
,7
]}

机构：

[1] Deakin Univ, Sch Med, 75 Pigdons Rd, Waurn Ponds, Vic 3216, Australia

[2] Deakin Univ, Ctr Mol & Med Res, 75 Pigdons Rd, Waurn Ponds, Vic 3216, Australia

[3] TCR2 Therapeut Inc, 100 Binney St, Cambridge, MA 02142 USA

[4] Univ Iowa, Internal Med, Iowa City, IA 52242 USA

[5] VP Platform Discovery Sci Biol Wave Life Sci, Cambridge, MA 02138 USA

[6] Natl Res Council CNR, Inst Genet & Biomed Res IRGB, UOS Milan Via Rita Levi Montalcini, I-20090 Milan, Italy

[7] Harvard Med Sch, Initiat RNA Med, Boston, MA 02115 USA

来源：

MOLECULAR THERAPY | 2021年 / 29卷 / 08期

关键词：

TARGETED DRUG-DELIVERY; BLOOD-BRAIN-BARRIER; IN-VITRO SELECTION; IPILIMUMAB PLUS DACARBAZINE; CELL ADHESION MOLECULE; SMALL ACTIVATING RNA; BIFUNCTIONAL APTAMER; TRANSFERRIN RECEPTOR; CURRENT CHALLENGES; GENE-EXPRESSION;

D O I：

10.1016/j.ymthe.2021.06.010

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

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 hardto-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.

引用

页码：2396 / 2411

页数：16

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