从罕见疾病患者的iPS细胞揭示衰老和癌症

tpwang

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) L0 ^6 F! j1 A4 v3 l/ l/ M去年的这个研究的潜在“意义”可以用文章结尾的讨论概括一下：
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（1）By reprogramming somatic cells from patients with the human disease dyskeratosis congenita, we have discovered novel mechanisms of regulation of telomerase activity in the pluripotent state, thus illustrating the value of disease-specific iPS cells for basic and translational discovery.
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成体细胞重编程为多潜能干细胞的过程修复了DC病的端粒酶缺陷，因此iPSC有可能用于治疗这类疾病。
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3 N: ?3 ^4 T6 t: e, Z（2）Moreover, we have shown that the RNA component of telomerase is upregulated in the pluripotent state to a degree sufficient to overcome limitations to telomere maintenance in X-linked and autosomal dominant DC. Drugs that activate the TERC locus may favour telomere maintenance over premature attrition in stemcell compartments where TERT is present but telomerase activity is limiting, such as haematopoietic stem cells23–27, and thus might serve as therapeutic agents for bone marrow failure.
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( |- }. v9 [$ X- C- B$ h激活TERC locus的药物可通过维持端粒酶活性而有利于骨髓移植。) W* o3 |# u' W. ?0 G- ?
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（3）Moreover, we speculate that in DC patients certain cell types such as germ cells and cancer cells, whose transcriptional programs share similarities with pluripotent cells, may also upregulate TERC to permit germline propagation of mutations and malignant proliferation.& x$ p) }4 |" o

/ ?! e( Y( |. P( K0 ~推断DC患者癌症扩散和恶化的原因可能是TERC的增高。( ~! ?) e$ g" C) N+ d7 h7 o

  z# L: P! F$ Y2 V+ H; c8 kiPSC可以延长端粒，恢复端粒酶活性，而且这与癌症状态相似，是不是也提示iPSC与癌症起码在端粒这点上是类似的。如果延长端粒或提升端粒酶活性有助于治疗某些早衰疾病尚可理解，但要说有可能开辟了抗衰老新途径，那么抗衰老的同时（通过改变端粒酶活性）是不是也同时增加了癌症的风险。就这项研究的三个所谓“意义”而言，如何在治疗这种疾病的同时避免提升癌症的风险，作者没有说有什么考虑。起码是留了个“自相矛盾”的尾巴。
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7 z) X& V8 h# z9 V) L文章是否以前发过了。7 h( N  \. C% z7 m; C. c