腺相关病毒
药物输送
医学
病毒学
生物
纳米技术
载体(分子生物学)
遗传学
材料科学
基因
重组DNA
作者
Tadatoshi Sato,Sachin Chaugule,Matthew B. Greenblatt,Guangping Gao,Jae‐Hyuck Shim
出处
期刊:Human Gene Therapy
[Mary Ann Liebert]
日期:2024-04-25
摘要
The development of bone-targeting drug delivery systems holds immense promise for improving the treatment of skeletal diseases. By precisely delivering therapeutic agents to the affected areas of bone, these strategies can enhance drug efficacy, minimize off-target effects, and promote patient adherence, ultimately leading to improved treatment outcomes and an enhanced quality of life for patients. This review aims to provide an overview of the current state of affinity-based bone-targeting agents and recent breakthroughs in innovative bone-targeting adeno-associated virus (AAV) strategies to treat skeletal diseases in mice. In particular, this review will delve into advanced AAV engineering, including AAV serotype selection for bone targeting and capsid modifications for bone-specific tropism. Additionally, we will highlight recent advancements in AAV-mediated gene therapy for skeletal diseases and discuss challenges and future directions of this promising therapeutic approach.
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