-
公开(公告)号：US20110262399A1
公开(公告)日：2011-10-27
申请号：US13121532
申请日：2009-09-29
申请人： Antonio Fontanellas Romá , Gloria González Aseguinolaza , Maria Sol Rodriguez Pena , Maria Astrid Pañeda Rodriguez , Jaap Twisk , Jesús Maria Prieto Valtueña , Harald Petry , Sander Jan Hendrik Van Deventer
发明人： Antonio Fontanellas Romá , Gloria González Aseguinolaza , Maria Sol Rodriguez Pena , Maria Astrid Pañeda Rodriguez , Jaap Twisk , Jesús Maria Prieto Valtueña , Harald Petry , Sander Jan Hendrik Van Deventer
CPC分类号： C12N9/88 , A61K38/00 , A61K48/0058 , C12N2799/025 , C12Y205/01061
摘要： The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.
摘要翻译： 本发明涉及编码针对哺乳动物细胞中较高表达的人胆色素原脱氨酶的核苷酸序列。 本发明还涉及包含用于基因治疗由胆毒素原脱氨酶（例如急性间歇性卟啉症）缺乏引起的病症的优化的合成编码序列的DNA构建体。 因此，本发明涉及包含编码人胆色素原脱氨酶的核苷酸序列的核酸或核酸构建体，其中至少320个编码人胆色素原脱氨酶的密码子与SEQ ID NO：1中的密码子相同 或其中至少305个编码人胆色素原脱氨酶的密码子与SEQ ID NO：3中的密码子相同。
-
公开(公告)号：US08697665B2
公开(公告)日：2014-04-15
申请号：US13121532
申请日：2009-09-29
申请人： Antonio Fontanellas Romá , Gloria González Aseguinolaza , Maria Sol Rodriguez Pena , Maria Astrid Pañeda Rodriguez , Jaap Twisk , Jesús Maria Prieto Valtueña , Harald Petry , Sander Jan Hendrik Van Deventer
发明人： Antonio Fontanellas Romá , Gloria González Aseguinolaza , Maria Sol Rodriguez Pena , Maria Astrid Pañeda Rodriguez , Jaap Twisk , Jesús Maria Prieto Valtueña , Harald Petry , Sander Jan Hendrik Van Deventer
CPC分类号： C12N9/88 , A61K38/00 , A61K48/0058 , C12N2799/025 , C12Y205/01061
摘要： The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.
摘要翻译： 本发明涉及编码针对哺乳动物细胞中更高表达的人胆色素原脱氨酶的核苷酸序列。 本发明还涉及包含用于基因治疗由胆毒素原脱氨酶（例如急性间歇性卟啉症）缺乏引起的病症的优化的合成编码序列的DNA构建体。 因此，本发明涉及包含编码人胆色素原脱氨酶的核苷酸序列的核酸或核酸构建体，其中至少320个编码人胆色素原脱氨酶的密码子与SEQ ID NO：1中的密码子相同 或其中至少305个编码人胆色素原脱氨酶的密码子与SEQ ID NO：3中的密码子相同。
-
搜索结果
2 条记录 (耗时 0.033 秒)
