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公开(公告)号：US08546143B2
公开(公告)日：2013-10-01
申请号：US12894018
申请日：2010-09-29
CPC分类号： C12N15/1138 , A61K38/00 , C12N15/111 , C12N15/1131 , C12N2310/14 , C12N2310/53 , C12N2320/50
摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.
摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。
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公开(公告)号：US20110111493A1
公开(公告)日：2011-05-12
申请号：US12894018
申请日：2010-09-29
CPC分类号： C12N15/1138 , A61K38/00 , C12N15/111 , C12N15/1131 , C12N2310/14 , C12N2310/53 , C12N2320/50
摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.
摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。
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公开(公告)号：US07829693B2
公开(公告)日：2010-11-09
申请号：US10384339
申请日：2003-03-07
CPC分类号： C12N15/1138 , A61K38/00 , C12N15/111 , C12N15/1131 , C12N2310/14 , C12N2310/53 , C12N2320/50
摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.
摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。
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