Epstein Benjamin E, Schaffer David V
Departments of Chemical and Biomolecular Engineering, Bioengineering, and Molecular and Cell Biology, University of California, Berkeley, 274B Stanley Hall, UC Berkeley, Berkeley, CA, 94720, USA.
Department of Bioengineering, University of California, Berkeley, 203 Stanley Hall, UC Berkeley, Berkeley, CA, 94720, USA.
Adv Exp Med Biol. 2017;1016:29-42. doi: 10.1007/978-3-319-63904-8_2.
With the recent advent of several generations of targeted DNA nucleases, most recently CRISPR/Cas9, genome editing has become broadly accessible across the biomedical community. Importantly, the capacity of these nucleases to modify specific genomic loci associated with human disease could render new classes of genetic disease, including autosomal dominant or even idiopathic disease, accessible to gene therapy. In parallel, the emergence of adeno-associated virus (AAV) as a clinically important vector raises the possibility of integrating these two technologies towards the development of gene editing therapies. Though clear challenges exist, numerous proof-of-concept studies in preclinical models offer exciting promise for the future of gene therapy.
随着几代靶向DNA核酸酶的出现，最近的CRISPR/Cas9，基因组编辑已在整个生物医学界广泛应用。重要的是，这些核酸酶修饰与人类疾病相关的特定基因组位点的能力，可能使包括常染色体显性甚至特发性疾病在内的新型遗传疾病能够接受基因治疗。同时，腺相关病毒（AAV）作为一种具有临床重要性的载体的出现，增加了将这两种技术整合用于基因编辑疗法开发的可能性。尽管存在明显的挑战，但临床前模型中的大量概念验证研究为基因治疗的未来带来了令人兴奋的前景。
