基因治疗近年来取得了实质性进展,成为治疗各种人类疾病的建设性策略。这篇综述全面概述了这些发展,专注于它们在不同疾病环境中的不同应用。它探索了基因传递系统的进化,包括病毒(如腺相关病毒;AAV)和非病毒方法,并评估其固有的优势和局限性。此外,该综述深入研究了在靶向特定组织和细胞类型方面取得的进展,跨越眼睛,肝脏,肌肉,和中枢神经系统,其中,利用这些基因技术。这种有针对性的方法对于解决广泛的遗传疾病至关重要,如遗传性溶酶体贮积病,神经退行性疾病,和心血管疾病。最近的临床试验和基因治疗的成功结果,特别是那些涉及AAV和成簇的规则间隔短回文重复(CRISPR)-CRISPR相关蛋白,被突出显示,阐明这种方法在疾病治疗中的转化潜力。综述了基因治疗的现状,其前景,及其显著改善患者预后和生活质量的能力。通过提供全面的分析,这篇综述为研究人员提供了宝贵的见解,临床医生,和利益相关者,丰富了正在进行的关于疾病治疗轨迹的话语。
Gene therapy has witnessed substantial advancements in recent years, becoming a constructive tactic for treating various human diseases. This review presents a comprehensive overview of these developments, with a focus on their diverse applications in different disease contexts. It explores the evolution of gene delivery systems, encompassing viral (like adeno-associated virus; AAV) and nonviral approaches, and evaluates their inherent strengths and limitations. Moreover, the review delves into the progress made in targeting specific tissues and cell types, spanning the eye, liver, muscles, and central nervous system, among others, using these gene technologies. This targeted approach is crucial in addressing a broad spectrum of genetic disorders, such as inherited lysosomal storage diseases, neurodegenerative disorders, and cardiovascular diseases. Recent clinical trials and successful outcomes in gene therapy, particularly those involving AAV and the clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated proteins, are highlighted, illuminating the transformative potentials of this approach in disease treatment. The review summarizes the current status of gene therapy, its prospects, and its capacity to significantly ameliorate patient outcomes and quality of life. By offering comprehensive analysis, this review provides invaluable insights for researchers, clinicians, and stakeholders, enriching the ongoing discourse on the trajectory of disease treatment.