Papanikolaou Eleni, Bosio Andreas
Department of Molecular Technologies and Stem Cell Therapy, Miltenyi Biotec, Bergisch Gladbach, Germany.
Laboratory of Biology, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece.
Front Genome Ed. 2021 Mar 24;3:618346. doi: 10.3389/fgeed.2021.618346. eCollection 2021.
It has been over 30 years since visionary scientists came up with the term "Gene Therapy," suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice.
自富有远见的科学家提出“基因治疗”这一术语以来，已经过去了30多年。这表明，对于某些适应症，主要是单基因疾病，通过基因添加用正常等位基因替代缺失或突变的基因，可以为受影响的患者提供持久的治疗效果，从而改善他们的生活质量。最近，这一概念已在某些疾病（如血红蛋白病和免疫缺陷以及其他单基因疾病）中成为现实。然而，基因治疗的浪潮不仅应用于这种情况，随着嵌合抗原受体T细胞（CAR-T）疗法的出现，它更广泛地用于治疗癌症。本综述将总结基因治疗从实验室到临床的逐步发展，主要关注造血干细胞基因治疗和基因组编辑，并将对基因治疗的未来及其在日常临床实践中的逐步整合提供一些有用的见解。
