CN116726035A — 用于包括线粒体dna耗竭综合症在内的由不平衡的核苷酸库引起的疾病的脱氧核苷疗法
Assigned to Fund Hospital University Vall D'hebron Institut De Recerca · Expires 2023-09-12 · 3y expired
What this patent protects
本发明通常涉及人类遗传疾病的药理学疗法,所述人类遗传疾病具体地是以不平衡的核苷酸库为特征的那些,更具体地是线粒体DNA耗竭综合症,且更具体地是胸苷激酶2(TK2)缺乏症。所述药理学疗法涉及施用至少一种脱氧核苷或其混合物。对于治疗TK2缺乏症,所述药理学疗法涉及施用脱氧胸苷(dT)或脱氧胞苷(dC)或其混合物。脱氧核苷的这种施用适用于不平衡的核苷酸库的其它病症,特别是见于线粒体DNA耗竭综合症的那些。
USPTO Abstract
本发明通常涉及人类遗传疾病的药理学疗法,所述人类遗传疾病具体地是以不平衡的核苷酸库为特征的那些,更具体地是线粒体DNA耗竭综合症,且更具体地是胸苷激酶2(TK2)缺乏症。所述药理学疗法涉及施用至少一种脱氧核苷或其混合物。对于治疗TK2缺乏症,所述药理学疗法涉及施用脱氧胸苷(dT)或脱氧胞苷(dC)或其混合物。脱氧核苷的这种施用适用于不平衡的核苷酸库的其它病症,特别是见于线粒体DNA耗竭综合症的那些。
Drugs covered by this patent
- Kygevvi (DOXECITINE) · Ucb Inc
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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