基于肽的载体:基因传递的生物分子工程策略。

IF 7.6 2区 工程技术 Q1 CHEMISTRY, APPLIED Annual review of chemical and biomolecular engineering Pub Date : 2023-06-08 Epub Date: 2023-03-08 DOI:10.1146/annurev-chembioeng-101121-070232
Sandeep Urandur, Millicent O Sullivan
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引用次数: 0

摘要

从W.F. Anderson博士的首次临床试验到最近美国食品药品管理局批准的Luxturna(Spark Therapeutics,2017年)和Zolgensma(诺华,2019年),基因疗法改变了癌症治疗的思路和实践,提高了成人和儿童遗传病患者的生存率。要将基因疗法应用于更广泛的领域,面临的一大挑战是如何安全地将核酸输送到预定的作用位点。肽具有与生物大分子和细胞相互作用的多样性和可调性,因此具有改善核酸递送的独特潜力。细胞穿透肽和细胞内靶向肽由于有望改善基因疗法向细胞内的递送而受到特别关注。我们将重点介绍肽辅助靶向基因递送到肿瘤生长的癌症特异性标志物和亚细胞器靶向肽的主要实例,以及提高肽稳定性和生物利用度以支持长期应用的新兴策略。
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Peptide-Based Vectors: A Biomolecular Engineering Strategy for Gene Delivery.

From the first clinical trial by Dr. W.F. Anderson to the most recent US Food and Drug Administration-approved Luxturna (Spark Therapeutics, 2017) and Zolgensma (Novartis, 2019), gene therapy has revamped thinking and practice around cancer treatment and improved survival rates for adult and pediatric patients with genetic diseases. A major challenge to advancing gene therapies for a broader array of applications lies in safely delivering nucleic acids to their intended sites of action. Peptides offer unique potential to improve nucleic acid delivery based on their versatile and tunable interactions with biomolecules and cells. Cell-penetrating peptides and intracellular targeting peptides have received particular focus due to their promise for improving the delivery of gene therapies into cells. We highlight key examples of peptide-assisted, targeted gene delivery to cancer-specific signatures involved in tumor growth and subcellular organelle-targeting peptides, as well as emerging strategies to enhance peptide stability and bioavailability that will support long-term implementation.

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来源期刊
Annual review of chemical and biomolecular engineering
Annual review of chemical and biomolecular engineering CHEMISTRY, APPLIED-ENGINEERING, CHEMICAL
CiteScore
16.00
自引率
0.00%
发文量
25
期刊介绍: The Annual Review of Chemical and Biomolecular Engineering aims to provide a perspective on the broad field of chemical (and related) engineering. The journal draws from disciplines as diverse as biology, physics, and engineering, with development of chemical products and processes as the unifying theme.
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