纳米技术与CRISPR/Cas介导的基因治疗策略:治疗遗传疾病的潜在作用。
Nanotechnology and CRISPR/Cas-Mediated Gene Therapy Strategies: Potential Role for Treating Genetic Disorders.
作者信息
Kaur Guneet, Arora Jasnoor, Sodhi Abhinashi Singh, Bhatia Sonu, Batra Navneet
机构信息
Department of Biotechnology, Goswami Ganesh Dutta Sanatan Dharma College, Sector-32-C, Chandigarh, 160030, India.
出版信息
Mol Biotechnol. 2024 Oct 24. doi: 10.1007/s12033-024-01301-8.
Gene therapy has made substantial progress in the treatment of the genetic diseases, focussing on the reduction of characteristics of recessive/dominant disorders, as well as various cancers. Extensive research has been conducted in the past few decades to investigate the application of nanotechnology and CRISPR/Cas technology in gene therapy. Nanotechnology due to attributes such has targeted drug delivery, controlled release, scalability and low toxicity has gained attention of the medical world. CRISPR/Cas9 system is considered as an impactful genome editing tool in the area of next-generation therapeutics and molecular diagnostics. CRISPR technology emphasises on gene editing, gene regulation modulation, and formulation of defined genetic changes. Its applications in treatment of the genetic disorders are extended beyond traditional therapies. These techniques are being explored as treatment of several genetic disorders including Duchenne muscular dystrophy, cystic fibrosis, Alzheimer's disease, Parkinson's disease, and Huntington disease. Despite considerable therapeutic potential of gene therapy, several obstacles must be addressed before it can be widely adopted in clinical practice, particularly in terms of ensuring safety and effectiveness. As research advances in this captivating field, these therapies will become the primary treatments and will have significant beneficial effects on the lives of patients with genetic disorders.
基因治疗在遗传疾病的治疗方面取得了重大进展,重点在于减少隐性/显性疾病的特征以及治疗各种癌症。在过去几十年里,人们进行了广泛的研究,以探讨纳米技术和CRISPR/Cas技术在基因治疗中的应用。由于具有靶向给药、控释、可扩展性和低毒性等特性,纳米技术受到了医学界的关注。CRISPR/Cas9系统被认为是下一代治疗学和分子诊断领域中一种有影响力的基因组编辑工具。CRISPR技术侧重于基因编辑、基因调控调节以及特定基因变化的形成。它在遗传疾病治疗中的应用超越了传统疗法。这些技术正在被探索用于治疗多种遗传疾病,包括杜氏肌营养不良症、囊性纤维化、阿尔茨海默病、帕金森病和亨廷顿病。尽管基因治疗具有相当大的治疗潜力,但在其能够在临床实践中广泛应用之前,必须解决几个障碍,特别是在确保安全性和有效性方面。随着这一引人入胜的领域的研究不断推进,这些疗法将成为主要治疗方法,并将对遗传疾病患者的生活产生重大有益影响。