Mulcrone Patrick L, Herzog Roland W, Xiao Weidong
Herman B Wells Center for Pediatric Research, Indiana University, Indianapolis, IN 46202, USA.
Department of Pediatrics, Indiana University, Indianapolis, IN 46202, USA.
Mol Ther Oncolytics. 2022 Oct 2;27:73-88. doi: 10.1016/j.omto.2022.09.009. eCollection 2022 Dec 15.
Gene therapy is a powerful biological tool that is reshaping therapeutic landscapes for several diseases. Researchers are using both non-viral and viral-based gene therapy methods with success in the lab and the clinic. In the cancer biology field, gene therapies are expanding treatment options and the possibility of favorable outcomes for patients. While cellular immunotherapies and oncolytic virotherapies have paved the way in cancer treatments based on genetic engineering, recombinant adeno-associated virus (rAAV), a viral-based module, is also emerging as a potential cancer therapeutic through its malleability, specificity, and broad application to common as well as rare tumor types, tumor microenvironments, and metastatic disease. A wide range of AAV serotypes, promoters, and transgenes have been successful at reducing tumor growth and burden in preclinical studies, suggesting more groundbreaking advances using rAAVs in cancer are on the horizon.
基因治疗是一种强大的生物学工具,正在重塑多种疾病的治疗格局。研究人员在实验室和临床中成功地使用了非病毒和基于病毒的基因治疗方法。在癌症生物学领域,基因治疗正在扩大治疗选择范围,并为患者带来良好预后的可能性。虽然细胞免疫疗法和溶瘤病毒疗法在基于基因工程的癌症治疗中开辟了道路,但基于病毒的模块重组腺相关病毒(rAAV)也因其可塑性、特异性以及对常见和罕见肿瘤类型、肿瘤微环境及转移性疾病的广泛适用性,而成为一种潜在的癌症治疗手段。在临床前研究中,多种AAV血清型、启动子和转基因已成功减少肿瘤生长和负担,这表明使用rAAV在癌症治疗方面将有更多突破性进展。