Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes.
Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes.
复制标题
用于治疗杜氏肌营养不良症及其他疾病的人类人工染色体：挑战和希望。
DOI：
10.1007/s10577-014-9460-6
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发表时间：
2015
期刊：
影响因子：
--
通讯作者：
Tedesco FS
中科院分区：
文献类型：
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作者：
Tedesco FS
安全有效、能够携带大量或多种转基因的载体是基因治疗的关键。人类人造染色体可以满足这一基本要求；此外，它们不会整合到宿主基因组中。然而，染色体转移效率低、工程相对复杂等缺点仍然限制了其广泛应用。在这篇文章中，我总结了将人类人工染色体带入杜氏肌营养不良症（一种x连锁的单基因疾病）临床前研究的关键步骤。我还将回顾这项技术未来可能的临床前和临床应用前景。
Safe and efficacious vectors able to carry large or several transgenes are of key importance for gene therapy. Human artificial chromosomes can fulfil this essential requirement; moreover, they do not integrate into the host genome. However, drawbacks such as the low efficiency of chromosome transfer and their relatively complex engineering still limit their widespread use. In this article, I summarise the key steps that brought human artificial chromosomes into preclinical research for Duchenne muscular dystrophy, an X-linked, monogenic disorder. I will also review possible future pre-clinical and clinical perspectives for this technology.