Adeno-Associated Virus Vectors-a Target of Cellular and Humoral Immunity-are Expanding Their Reach Toward Hematopoietic Stem Cell Modification and Immunotherapies.

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Human gene therapy Pub Date : 2024-09-01 Epub Date: 2024-08-28 DOI:10.1089/hum.2024.114
Angela E Araujo, Martin Bentler, Xabier Perez Garmendia, Asma Kaleem, Claire Fabian, Michael Morgan, Ulrich T Hacker, Hildegard Büning
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引用次数: 0

Abstract

All current market-approved gene therapy medical products for in vivo gene therapy of monogenic diseases rely on adeno-associated virus (AAV) vectors. Advances in gene editing technologies and vector engineering have expanded the spectrum of target cells and, thus, diseases that can be addressed. Consequently, AAV vectors are now being explored to modify cells of the hematopoietic system, including hematopoietic stem and progenitor cells (HSPCs), to develop novel strategies to treat monogenic diseases, but also to generate cell- and vaccine-based immunotherapies. However, the cell types that represent important new targets for the AAV vector system are centrally involved in immune responses against the vector and its transgene product as discussed briefly in the first part of this review. In the second part, studies exploring AAV vectors for genetic engineering of HSPCs, T and B lymphocytes, and beyond are presented.

腺相关病毒载体--细胞免疫和体液免疫的靶标--正在向造血干细胞改造和免疫疗法领域拓展。
目前市场上批准用于单基因疾病体内基因治疗的所有基因治疗医疗产品都依赖于腺相关病毒(AAV)载体。基因编辑技术和载体工程学的进步扩大了靶细胞的范围,从而也扩大了可治疗疾病的范围。因此,AAV 载体目前正被用于改造造血系统细胞,包括造血干细胞和祖细胞(HSPCs),以开发治疗单基因疾病的新策略,同时也用于产生以细胞和疫苗为基础的免疫疗法。然而,AAV载体系统的重要新靶点细胞类型主要参与了针对载体及其转基因产物的免疫反应,本综述的第一部分对此进行了简要讨论。第二部分将介绍探索 AAV 载体用于 HSPC、T 淋巴细胞和 B 淋巴细胞等基因工程的研究。
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来源期刊
Human gene therapy
Human gene therapy 医学-生物工程与应用微生物
CiteScore
6.50
自引率
4.80%
发文量
131
审稿时长
4-8 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
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