New recombinant adeno-associated virus vector comprising a nucleic acid sequence encoding an optimized retinal pigment epithelium 65 kDa protein transgene with a nucleotide sequence, used in dual-vector system for gene therapy of leber congenital amaurosis type 2 and other retinal disorders
	KUMAR N G; HUDA S; SINGH V; RAO J G
	2024-05-15
专利权人	INDIAN INST TECHNOLOGY KANPUR (IITK-C)
申请日期	2024-05-15
专利号	IN202411038240-A
成果简介	NOVELTY - Recombinant adeno-associated virus (AAV) vector is new, where AAV vector comprises a nucleic acid sequence encoding an optimized retinal pigment epithelium 65 kDa protein (RPE65) transgene with a nucleotide sequence comprising 6389 nucleotides (SEQ ID NO: 1), given in the specification, wherein the optimized RPE65 transgene is a codon-optimized RPE65 transgene designed to optimise gene expression and inhibit apoptosis in retinal cells, and wherein the optimized RPE65 transgene is under control of a cytomegalovirus (CMV) promoter-enhancer system, and a nucleic acid sequence encoding an anti-apoptotic factor with a nucleotide sequence comprising 5476 nucleotide (SEQ ID NO: 2), given in the specification, where the anti-apoptotic factor is survivin transgene. USE - Recombinant adeno-associated virus vector used in dual-vector system for gene therapy of leber congenital amaurosis type 2 (LCA2) and other retinal disorders. No biological data given. ADVANTAGE - The codon25 optimised RPE65 gene is modified to optimise translational efficiency and protein stability in retinal cells. The first AAV vector and second AAV vector are of different serotypes to minimize interference between transgenes. The combination of the optimized RPE65 transgene and the survivin gene therapy reduces apoptosis of photoreceptors and retinal pigment epithelial (RPE) cells. The AAV vector enhances therapeutic efficacy of gene replacement therapy targeting a RPE65 gene, as well as the co delivery of anti-apoptotic factors such as survivin, aimed at slowing retinal degeneration and sustaining long-term vision rescue, and provide strategy for improving outcomes in LCA2 and other retinal disorders, potentially enhancing patient quality of life. DETAILED DESCRIPTION - INDEPENDENT CLAIMS are included for: Dual-vector system for gene therapy of leber congenital amaurosis type 2 (LCA2), which comprises a first AAV vector encoding an optimized RPE65 transgene with a nucleotide sequence SEQ ID NO: 1, and a second AAV vector encoding an anti-apoptotic factor with a nucleotide sequence SEQ ID NO: 2, where the anti-apoptotic factor is survivin transgene; and Method for producing a recombinant adeno-associated virus (AAV) vector for encoding an optimized RPE65 transgene.
IPC 分类号	A61K-048/00 ; A61P-027/02 ; C12N-015/66 ; C12N-015/86 ; C12N-015/861
国家	印度
专业领域	医药卫生
语种	英语
成果类型	专利
文献类型	科技成果
条目标识符	http://119.78.100.226:8889/handle/3KE4DYBR/16762
专题	中国科学院新疆生态与地理研究所
作者单位	INDIAN INST TECHNOLOGY KANPUR (IITK-C)
推荐引用方式 GB/T 7714	KUMAR N G,HUDA S,SINGH V,et al. New recombinant adeno-associated virus vector comprising a nucleic acid sequence encoding an optimized retinal pigment epithelium 65 kDa protein transgene with a nucleotide sequence, used in dual-vector system for gene therapy of leber congenital amaurosis type 2 and other retinal disorders. IN202411038240-A[P]. 2024.

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