{"title":"基于肽的载体:基因传递的生物分子工程策略。","authors":"Sandeep Urandur, Millicent O Sullivan","doi":"10.1146/annurev-chembioeng-101121-070232","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":8234,"journal":{"name":"Annual review of chemical and biomolecular engineering","volume":"14 ","pages":"243-264"},"PeriodicalIF":7.6000,"publicationDate":"2023-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Peptide-Based Vectors: A Biomolecular Engineering Strategy for Gene Delivery.\",\"authors\":\"Sandeep Urandur, Millicent O Sullivan\",\"doi\":\"10.1146/annurev-chembioeng-101121-070232\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":8234,\"journal\":{\"name\":\"Annual review of chemical and biomolecular engineering\",\"volume\":\"14 \",\"pages\":\"243-264\"},\"PeriodicalIF\":7.6000,\"publicationDate\":\"2023-06-08\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Annual review of chemical and biomolecular engineering\",\"FirstCategoryId\":\"5\",\"ListUrlMain\":\"https://doi.org/10.1146/annurev-chembioeng-101121-070232\",\"RegionNum\":2,\"RegionCategory\":\"工程技术\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2023/3/8 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q1\",\"JCRName\":\"CHEMISTRY, APPLIED\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Annual review of chemical and biomolecular engineering","FirstCategoryId":"5","ListUrlMain":"https://doi.org/10.1146/annurev-chembioeng-101121-070232","RegionNum":2,"RegionCategory":"工程技术","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2023/3/8 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"CHEMISTRY, APPLIED","Score":null,"Total":0}
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.
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.