Nat Biotechnol. 2011 Feb;29(2):121-8. doi: 10.1038/nbt.1769.
Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens.
基因治疗终于有望为治疗那些其他疗法无法解决的、具有高度外显率的、使人虚弱的遗传疾病做出贡献。
