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端粒亦称端区，位于真核细胞染色体末端，随细胞分裂而脱失，缩短至一定程度时体细胞死亡，癌细胞因具有合成端粒的端粒酶而获永生。端粒酶亦称端聚酶，为一含ＲＮＡ的核糖核蛋白，在９０％的癌细胞中过表达，成为肿瘤治疗的新靶点。以端粒酶为靶点的药物设计策略包括端粒酶ＲＮＡ和编码蛋白质的基因以及编码端粒结合蛋白基因的操作。另外，染色体转移、诱导分化、细胞周期调节及常规药物筛选亦为重要的策略。其中针对端粒酶ＲＮＡ模板区的反义（寡）核（苷）酸或Ｒｉｂｏｚｙｍｅ及其突变体的设计构成基因治疗的主体。
The telomere, also known as the terminal region, is located at the end of chromosomes of eukaryotic cells, and it desorbs with cell division. When it is shortened to a certain extent, somatic cells die. The cancer cells are immortalized by telomerase that synthesizes telomeres. Telomerase, also known as telomerase, is an RNA-containing ribonucleoprotein that is overexpressed in 90% of cancer cells and is a new target for cancer therapy. Telomerase-targeted drug design strategies include telomerase RNA and genes encoding proteins and manipulations of genes encoding telomere binding proteins. In addition, chromosome transfer, induced differentiation, cell cycle regulation, and routine drug screening are also important strategies. The design of antisense (oligo)nucleotide (glycosamino) acid or Ribozyme and its mutants against the telomerase RNA template region constitute the main body of gene therapy.