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AAV VECTORS FOR THE TREATMENT OF ISCHEMIC AND NON-ISCHEMIC HEART DISEASE

机译:AAV矢量治疗缺血性和非缺血性心脏病

摘要

The present invention refers to the biotechnological field, more particularly to the use of the calcineurin subunit Αβ1 isoform (CnΑβ1) for the treatment or prevention of ischemic and non¬ ischemic heart disease. Specifically, the present invention refers to a virion-free nucleic acid construct comprising a nucleotide sequence selected from the list consisting of: a. A nucleic acid sequence encoding SEQ ID No 1, or the complementary sequence of said nucleic acid sequence; b. a nucleic sequence encoding a peptide comprising a fragment of SEQ ID No 1, wherein said fragment consists of SEQ ID No 2, or the complementary sequence of said nucleic acid sequence; or c. a variant of a) or b) which encodes a peptide which is at least 80% homologous to SEQ ID No 1 or SEQ ID No 2 based on amino acid identity; wherein the nucleotide sequence is operably linked to control elements which direct the transcription and translation of the nucleotide sequence and wherein the nucleotide sequence operably linked to the control elements is flanked by a 5' and a 3' adeno-associated virus inverted terminal repeat (AAV ITRs).
机译:本发明涉及生物技术领域,更具体地涉及钙调神经磷酸酶亚基Aβ1同工型(CnAβ1)在治疗或预防缺血性和非缺血性心脏病中的用途。具体地,本发明涉及包含选自以下的核苷酸序列的无病毒体核酸构建体:a。编码SEQ ID No 1的核酸序列,或所述核酸序列的互补序列; b。编码包含SEQ ID No 1的片段的肽的核酸序列,其中所述片段由SEQ ID No 2或所述核酸序列的互补序列组成;或c。 a)或b)的变体,其编码基于氨基酸同一性与SEQ ID No 1或SEQ ID No 2至少80%同源的肽;其中所述核苷酸序列与控制元件可操作地连接,所述控制元件指导所述核苷酸序列的转录和翻译,并且其中与所述控制元件可操作地连接的所述核苷酸序列的侧翼是5'和3'腺相关病毒反向末端重复序列(AAV) ITR)。

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