Madrigal Justin L, Stilhano Roberta, Silva Eduardo A
Department of Biomedical Engineering, University of California , Davis, Davis, California.
Tissue Eng Part B Rev. 2017 Aug;23(4):347-361. doi: 10.1089/ten.TEB.2016.0462. Epub 2017 Mar 10.
Gene therapy is a promising strategy for musculoskeletal tissue repair and regeneration where local and sustained expression of proteins and/or therapeutic nucleic acids can be achieved. However, the musculoskeletal tissues present unique engineering and biological challenges as recipients of genetic vectors. Targeting specific cell populations, regulating expression in vivo, and overcoming the harsh environment of damaged tissue accompany the general concerns of safety and efficacy common to all applications of gene therapy. In this review, we will first summarize these challenges and then discuss how biomaterial carriers for genetic vectors can address these issues. Second, we will review how limitations specific to given vectors further motivate the utility of biomaterial carriers. Finally, we will discuss how these concepts have been combined with tissue engineering strategies and approaches to improve the delivery of these vectors for musculoskeletal tissue regeneration.
基因治疗是一种用于肌肉骨骼组织修复和再生的有前景的策略,通过该策略可实现蛋白质和/或治疗性核酸的局部和持续表达。然而,肌肉骨骼组织作为基因载体的接受者,存在独特的工程学和生物学挑战。靶向特定细胞群体、在体内调节表达以及克服受损组织的恶劣环境,伴随着基因治疗所有应用中常见的安全性和有效性问题。在本综述中,我们将首先总结这些挑战,然后讨论基因载体的生物材料载体如何解决这些问题。其次,我们将回顾特定载体的局限性如何进一步推动生物材料载体的应用。最后,我们将讨论这些概念如何与组织工程策略和方法相结合,以改善这些载体用于肌肉骨骼组织再生的递送。