Molecular medicine - Antisense oligonucleotide therapy

Molecular medicine - Antisense oligonucleotide therapy
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DOI:
10.1056/nejm199602013340508
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发表时间:
1996-02-01
影响因子:
158.5
通讯作者:
McDonnell, WM
McDonnell, WM
中科院分区:
医学1区
文献类型:
--
作者:
Askari, FK;McDonnell, WM

文献摘要

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许多药理学的进展涉及创造结合和禁用蛋白质的化合物。这些化合物包括普萘洛尔,其阻断β-肾上腺素能受体;西咪替丁,其阻断H2受体;钙通道阻断剂;血管紧张素转化酶抑制剂;和H +/K +-ATP酶泵抑制剂。阻断蛋白质功能的一种新方法是阻止信使RNA(mRNA)翻译成蛋白质。如果编码蛋白质的核酸序列已知,则可以设计分子来结合相应的mRNA,从而抑制蛋白质的产生(图1)。DNA或RNA的核苷酸序列,含有氨基酸序列的信息。
Many pharmacologic advances involve creating compounds that bind and disable proteins. Such compounds include propranolol, which blocks the β-adrenergic receptor; cimetidine, which blocks the H2receptor; calcium-channel blockers; angiotensin-converting–enzyme inhibitors; and inhibitors of the H+/K+–ATPase pump. A new way to block protein function is to prevent the translation of messenger RNA (mRNA) into protein.If the nucleic acid sequence encoding a protein is known, molecules can be designed to bind the corresponding mRNA, thereby inhibiting the production of the protein (Figure 1). The nucleotide sequence of DNA or RNA that contains the information for the amino acid sequence of . . .