Prabhakar P, Kayastha A M
School of Biotechnology, Faculty of Science, Banaras Hindu University, Varanasi, India.
Appl Biochem Biotechnol. 1994 Apr;47(1):39-55. doi: 10.1007/BF02788674.
Over the last two decades many strategies have been planned to design specific drugs for rare diseases to target their action at the DNA level. Advancements in our understanding of the interactions of small nonpeptide molecules with DNA have opened the doors for "rational" drug design. Special methods have now been developed to give accurate account of the precise location of ligand-DNA adducts on target DNA. We are now in a position to think of designing ligands that recognize particular sequences of base pairs. This work will allow us to enter into a new era of gene therapy for diseases like Cystic fibrosis, Alzheimer's disease and many related disorders at genetic level. These ligands can also be employed in the treatment of various types of cancers. They may also be useful as highly specific probes to locate particular sequences in the genomic DNA.
在过去二十年里,人们制定了许多策略来设计针对罕见病的特定药物,使其作用于DNA水平。我们对小分子非肽分子与DNA相互作用的理解取得了进展,为“理性”药物设计打开了大门。现在已经开发出特殊方法,能够准确描述配体-DNA加合物在靶DNA上的精确位置。我们现在能够考虑设计识别特定碱基对序列的配体。这项工作将使我们能够在基因层面进入针对囊性纤维化、阿尔茨海默病及许多相关疾病的基因治疗新时代。这些配体还可用于治疗各种类型的癌症。它们也可能作为高度特异性探针来定位基因组DNA中的特定序列。