Department of Microbiology, Immunology, and Molecular Genetics, and Department of Pediatrics, David Geffen School of Medicine, and Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA;
Department of Pediatrics, Stanford University, Stanford, CA;
Blood. 2016 May 26;127(21):2553-60. doi: 10.1182/blood-2016-01-678136. Epub 2016 Apr 6.
Gene editing is a rapidly developing area of biotechnology in which the nucleotide sequence of the genome of living cells is precisely changed. The use of genome-editing technologies to modify various types of blood cells, including hematopoietic stem cells, has emerged as an important field of therapeutic development for hematopoietic disease. Although these technologies offer the potential for generation of transformative therapies for patients suffering from myriad disorders of hematopoiesis, their application for therapeutic modification of primary human cells is still in its infancy. Consequently, development of ethical and regulatory frameworks that ensure their safe and effective use is an increasingly important consideration. Here, we review a number of issues that have the potential to impact the clinical implementation of genome-editing technologies, and suggest paths forward for resolving them such that new therapies can be safely and rapidly translated to the clinic.
基因编辑是生物技术中一个快速发展的领域，在此领域中，活细胞基因组的核苷酸序列可以被精确地改变。利用基因组编辑技术来修饰各种类型的血细胞，包括造血干细胞，已经成为造血疾病治疗开发的一个重要领域。尽管这些技术为患有各种造血障碍的患者带来了变革性治疗的潜力，但它们在原发性人类细胞的治疗修饰中的应用仍处于起步阶段。因此，制定确保其安全有效使用的伦理和监管框架是一个日益重要的考虑因素。在这里，我们回顾了一些有可能影响基因组编辑技术临床应用的问题，并为解决这些问题提出了一些建议，以便新的治疗方法能够安全、快速地推向临床。
