首页> 外国专利> Peptide-modified hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced hepatic tropism and increased muscle transduction

Peptide-modified hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced hepatic tropism and increased muscle transduction

机译:AAV9和AAVRH74之间的肽改性的杂交重组腺相关病毒血清型,其肝脏抗肝脏抗性和肌肉转导增加

摘要

The present invention relates to a recombinant adeno-associated virus (AAV) capsid protein that is a peptide-modified hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins comprising at least one copy of a peptide comprising RGD. to which the recombinant peptide-modified hybrid AAV capsid protein has more reduced hepatic tropism and increased muscle transduction compared to the recombinant hybrid AAV capsid protein without the peptide. The present invention also relates to derivatized peptide-modified hybrid AAV serotype vector particles packaging a gene of interest and their use for gene therapy, in particular for treating neuromuscular genetic diseases, in particular muscular genetic diseases.
机译:本发明涉及一种重组腺相关病毒(AAV)衣壳蛋白,其是AAV血清型9(AAV9)和AAV血清型74(AAVRH74)衣壳蛋白质之间的肽改性杂交蛋白,其包含包含RGD的肽的至少一种拷贝。 与没有肽的重组杂交AAV胶囊蛋白相比,重组肽改性的杂交AAV辣椒屑蛋白具有更高的肝脏抗皱蛋白和肌肉转移增加。 本发明还涉及衍生化的肽改性的杂交AAV血清型载体载体染色感兴趣的基因及其用于基因治疗的用途,特别是用于治疗神经肌肉遗传疾病,特别是肌肉遗传疾病。

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