Genome Engineering and Model Development lab (GEMD), IUF-Leibniz Research Institute for Environmental Medicine, 40225 Düsseldorf, Germany.
Front Biosci (Landmark Ed). 2022 Aug 12;27(8):241. doi: 10.31083/j.fbl2708241.
Genomic mutations are the driving force of biological diversity but they are also the cause of a plethora of human diseases ranging from heritable disorders to neurological pathologies and cancer. For most genetic disorders, there is no curative treatment available to date. The demand for precise, preferably patient-specific, treatment regimen offering cure is naturally high. Genome editing by Zinc Finger Nucleases (ZFNs), Transcription Activator-Like Effector Nucleases (TALENs), and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas enables targeted manipulation of genomes, thereby offering the opportunity to treat such diseases. While ethical and regulatory guidelines need to be developed and considered, the prospect of genome editing for curative treatment is certainly exciting. Here, we review the current state of therapeutics based on genome editing techniques. We highlight recent breakthroughs, describe clinical trials employing genome editing-based medicine, discuss the benefits and pitfalls, and take a look into the future of genome editing.
基因组突变是生物多样性的驱动力，但也是许多人类疾病的原因，包括遗传性疾病、神经病理学和癌症。对于大多数遗传疾病，目前尚无有效的治疗方法。因此，人们自然非常需要精确的、最好是针对患者个体的治疗方案来实现治愈。锌指核酸酶（ZFNs）、转录激活因子样效应核酸酶（TALENs）和规律成簇间隔短回文重复（CRISPR）/Cas 基因组编辑技术可实现基因组的靶向操作，从而为治疗此类疾病提供了机会。虽然需要制定和考虑伦理和监管准则，但基因组编辑用于治疗的前景无疑令人兴奋。在这里，我们回顾了基于基因组编辑技术的治疗现状。我们强调了最近的突破，描述了使用基于基因组编辑的医学的临床试验，讨论了其益处和风险，并展望了基因组编辑的未来。
