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一种治疗赫勒氏综合征的方法和药物

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摘要

一种基于LEAPER技术的核酸药物，及使用该核酸药物靶向编辑RNA治疗疾病例如赫勒氏综合征(Hurler syndrome)的方法。该方法包括使用所述核酸药物进行RNA上由腺苷到次黄嘌呤碱基的编辑，精准地修复例如赫勒氏综合征(Hurler syndrome)的致病性GA突变位点，恢复由RNA编码的蛋白例如IDUA在体内的正常表达。

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公开/公告号CN115697420A

专利类型发明专利
公开/公告日2023-02-03

原文格式PDF
申请/专利权人北京辑因医疗科技有限公司;
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申请/专利号CN202180028449.7
发明设计人袁鹏飞;赵艳霞;刘能银;易泽轩;汤刚彬;
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申请日2021-04-15
分类号A61K48/00;C12N15/113;C12N15/11;C12N15/88;A61P3/00;
代理机构北京彩和律师事务所;
代理人张红春
地址 102206 北京市昌平区科学园路22号2幢2层
入库时间 2023-06-19 18:32:25

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法律状态公告日

法律状态信息

法律状态
2023-02-03

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PCT国内申请，说明书已公开。

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1. 一种治疗柯兴氏综合征的内服药物及其制备方法 [P] . 中国专利： CN104306680A . 2015-01-28
2. 一种治疗雷诺氏综合征的汤剂药物及制备方法 [P] . 中国专利： CN104474062A . 2015-04-01
3. compound of formula i; pharmaceutical composition; method of treating disorders that are related to or affected by inhibition of norepinephrine reuptake, the 5-ht6 receptor or the 5-ht2a receptor; a method of treating a learning disorder, a cognitive disorder, a memory disorder, a personality disorder, a behavioral disorder, a movement disorder, a neurodegenerative disorder, drug withdrawal disorder, sleep, an eating disorder, acute drug toxicity, a cardiovascular disorder, a sexual dysfunction, a gastrointestinal disorder, a genitourinary disorder, a pain disorder, a nervous disorder or a symptom disorder vasomotor; method of treating alzheimer's disease, attention deficit disorder, schizophrenia, parkinson's disease, tardive dyskinesia, ataxia, bradykinesia, paroxysmal dyskinesias, restless leg syndrome, tremor, essential tremor epilepsy, stroke or head trauma; method of treating coronary artery disease, myocardial infarction, transient ischemic attack, angina, atrial fibrillation, p [P] . BRPI0706523A2 . 2011-01-04

机译：式I化合物;药物组合物;治疗与去甲肾上腺素再摄取，5-ht6受体或5-ht2a受体相关或受其抑制的疾病的方法;一种治疗学习障碍，认知障碍，记忆障碍，人格障碍，行为障碍，运动障碍，神经退行性疾病，戒断药物，睡眠，进食障碍，急性药物毒性，心血管疾病的方法，性功能障碍，胃肠道疾病，泌尿生殖系统疾病，疼痛疾病，神经疾病或症状-血管舒缩;治疗阿尔茨海默氏病，注意力缺陷障碍，精神分裂症，帕金森氏病，迟发性运动障碍，共济失调，运动迟缓，阵发性运动障碍，不安腿综合征，震颤，原发性震颤癫痫，中风或头部外伤的方法;治疗冠状动脉疾病，心肌梗塞，短暂性脑缺血发作，心绞痛，心房颤动，p的方法
4. compound, pharmaceutical composition; use of the compound for the manufacture of a medicament for treating irritable bowel syndrome, treating pain or painful syndromes, treating urinary incontinence, treating depression, fibromyalgia, anxiety, panic disorder, agorophobia, pre-traumatic stress disorder, pre-dysphoric disorder menstrual disorder, attention deficit disorder, obsessive compulsive disorder, social anxiety disorder, generalized anxiety disorder, autism, schizophrenia, obesity, anorexia nervosa, bulimia nervosa, gilles de la tourette syndrome, vasomotor discharge, cocaine addiction, sexual dysfunction, personality threshold disorder, fibromyalgia syndrome, diabetic neuropathic pain, chronic fatigue syndrome, shy drager syndrome, raynaud syndrome, parkinson's disease, and epilepsy, method of preparing a compound of structure (a), and Method of preparing a compound of structure (b) [P] . BRPI0613031A2 . 2012-01-03

机译：化合物，药物组合物;该化合物在制备用于治疗肠易激综合症，治疗疼痛或疼痛综合症，治疗尿失禁，治疗抑郁症，纤维肌痛，焦虑症，恐慌症，广场恐惧症，创伤前应激障碍，烦躁不安症，月经失调的药物中的用途，注意力缺陷障碍，强迫症，社交焦虑症，广泛性焦虑症，自闭症，精神分裂症，肥胖症，神经性厌食症，神经性贪食症，抽动秽语综合征，血管舒缩放电，可卡因成瘾，性功能障碍，人格阈障碍，纤维肌痛综合征，糖尿病性神经性疼痛，慢性疲劳综合症，害羞药物综合征，雷诺氏综合症，帕金森氏病和癫痫病，结构式（a）的化合物的制备方法和结构式（b）的化合物的制备方法
5. compound, pharmaceutical composition, method of selective activation/stimulation of nicotinic receptors from to-7 in a patient in which such activation/stimulation has a therapeutic effect, method of treating a patient suffering from a psychotic disease, a neurodegenerative disease involving a dysfunction of the cholinergic system, and/or a condition of impaired memory and/or cognition, method of treatment of a patient suffering from dementia and/or another condition with memory loss, method of treatment of a patient suffering from memory impairment due to mild cognitive impairment due to aging, alzheimer's malady,schizophrenia, parkinson malady, huntington malady, pick malady, creutzfeldt-jakob malady, depression, aging, head trauma, stroke, snc hypoxia, cerebral senility, multiinfarction dementia, HIV and/or cardiovascular disease, method of treatment and/or prevention of dementia in a patient with alzheimer's disease, method of treating a patient to cut off the alcohol vein or treat [P] . BRPI0517494A . 2008-10-14

机译：化合物，药物组合物，在其中这种激活/刺激具有治疗作用的患者中选择性激活/刺激烟碱受体从to-7的方法，治疗患有精神病，涉及神经功能障碍的神经退行性疾病的患者的方法胆碱能系统和/或记忆力和/或认知障碍的状况，患有痴呆症的患者的治疗方法和/或患有记忆力丧失的另一种状况，由于轻度认知障碍而遭受记忆力障碍的患者的治疗方法由于衰老，阿尔茨海默氏病，精神分裂症，帕金森氏病，亨廷顿氏病，匹克病，creutzfeldt-jakob病，抑郁症，衰老，头部外伤，中风，snc缺氧，脑衰老，多发性梗塞性痴呆，HIV和/或心血管疾病，治疗方法治疗和/或预防老年痴呆症患者的痴呆症，治疗患者的方法以切断酒精静脉或治疗