Harnessing the Therapeutic Potential of Biomacromolecules through Intracellular Delivery of Nucleic Acids, Peptides, and Proteins.
Harnessing the Therapeutic Potential of Biomacromolecules through Intracellular Delivery of Nucleic Acids, Peptides, and Proteins.
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DOI：
10.1002/adhm.202102600
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发表时间：
2022-06
影响因子：
10
通讯作者：
LaBelle, James L.
中科院分区：
文献类型：
--
作者：
Tian, Yu;Tirrell, Matthew, V;LaBelle, James L.
生物大分子长期以来一直处于学术和制药药物开发和临床转化的前沿。随着单克隆抗体和核酸等新疗法的临床进展，生物大分子的医学应用范围大大拓宽。一个主要的正在进行的努力是扩大细胞内的位置的治疗目标。由于生物大分子具有大的尺寸、丰富的电荷和氢键给体和受体，需要先进的递送技术来将生物大分子有效地递送到细胞内。在这篇综述中，我们强调用于细胞内递送三种主要形式的生物大分子：核酸，蛋白质和肽的策略。重点放在合成交付方法和需要克服的主要障碍，其最终的临床翻译。目前药物研究的趋势之一是利用传统上被认为是不可药用的细胞内靶点。新的合成递送方法使生物大分子能够接近这些细胞内靶点进行治疗干预。这些方法和局限性的例子进行了讨论，在这次审查。
Biomacromolecules have long been at the leading edge of academic and pharmaceutical drug development and clinical translation. With the clinical advances of new therapeutics, such as monoclonal antibodies and nucleic acids, the array of medical applications of biomacromolecules has broadened considerably. A major on-going effort is to expand therapeutic targets within intracellular locations. Owing to their large sizes, abundant charges, and hydrogen-bond donors and acceptors, advanced delivery technologies are required to deliver biomacromolecules effectively inside cells. In this review, we highlight strategies used for the intracellular delivery of three major forms of biomacromolecules: nucleic acids, proteins and peptides. An emphasis is placed on synthetic delivery approaches and the major hurdles needed to be overcome for their ultimate clinical translation. One of the current trends in drug research is to exploit the intracellular targets that have been traditionally considered undruggable. Novel, synthetic delivery approaches have enabled biomacromolecules to access these intracellular targets for therapeutic intervention. Examples of these approaches and limitations are discussed in this review.
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影响因子：
46.2
作者：
Behzadi S;Serpooshan V;Tao W;Hamaly MA;Alkawareek MY;Dreaden EC;Brown D;Alkilany AM;Farokhzad OC;Mahmoudi M
通讯作者：
Mahmoudi M
影响因子：
13.3
作者：
Akar, Ugur;Chaves-Reyez, Arturo;Ozpolat, Bulent
通讯作者：
Ozpolat, Bulent
DOI：
10.1073/pnas.0509035102
发表时间：
2005-12-13
影响因子：
11.1
作者：
Austin, CD;Wen, XH;Scales, SJ
通讯作者：
Scales, SJ
影响因子：
--
作者：
Arkin MR;Tang Y;Wells JA
通讯作者：
Wells JA
影响因子：
38.3
作者：
Akinc, Akin;Maier, Martin A.;Cullis, Pieter R.
通讯作者：
Cullis, Pieter R.