利索-全球专利检索

  1. 登录
  2. 注册

搜索结果

12 条记录 (耗时 0.017 秒)

    FABRY DISEASE GENE THERAPY
    1.
    发明申请
    FABRY DISEASE GENE THERAPY 审中-公开
    纤维蛋白病基因治疗

    公开(公告)号：WO2016181122A1

    公开(公告)日：2016-11-17

    申请号：PCT/GB2016/051328

    申请日：2016-05-10

    申请人： UCL BUSINESS PLC

    发明人： NATHWANI, Amit ,  RAJ, Deepak

    IPC分类号： A61K48/00 ,  C12N9/40 ,  C12N15/67 ,  C12N15/864

    CPC分类号： A61K48/0058 ,  A01K2217/075 ,  A61K38/47 ,  A61P3/00 ,  C12N9/2465 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2800/22

    摘要： There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional α-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.

    摘要翻译： 描述了包含编码功能性α-半乳糖苷酶A蛋白的核苷酸序列的核酸分子，其中所述核苷酸序列与SEQ ID NO的序列具有至少85％的同一性。 还描述了包含核酸分子的载体，宿主细胞或转基因动物; 和包含核酸分子或载体的药物组合物。 此外，描述了在治疗法布里病的方法中使用核酸分子。

    FACTOR IX GENE THERAPY
    2.
    发明申请
    FACTOR IX GENE THERAPY 审中-公开

    公开(公告)号：WO2016075473A3

    公开(公告)日：2016-05-19

    申请号：PCT/GB2015/053438

    申请日：2015-11-12

    申请人： UCL BUSINESS PLC

    发明人： NATHWANI, Amit ,  MCINTOSH, Jenny ,  PATEL, Nishil

    IPC分类号： C12N9/64

    摘要： The invention relates to a new, more potent, coagulation factor IX (FIX) expression cassette for gene therapy of haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.

    FACTOR IX ENCODING NUCLEOTIDES
    3.
    发明申请
    FACTOR IX ENCODING NUCLEOTIDES 审中-公开

    公开(公告)号：WO2020039183A1

    公开(公告)日：2020-02-27

    申请号：PCT/GB2019/052339

    申请日：2019-08-20

    申请人： UCL BUSINESS PLC

    发明人： NATHWANI, Amit ,  MCINTOSH, Jenny

    IPC分类号： C12N9/64 ,  A61P7/04 ,  C12N15/864 ,  A61K38/48

    摘要： The present invention relates to polynucleotides comprising a Factor IX nucleotide sequence, wherein the Factor IX nucleotide sequence comprises a coding sequence that encodes a Factor IX protein or fragment thereof and wherein a portion of the coding sequence is not wild type. The present invention further relates to viral particles comprising a recombinant genome comprising the polynucleotide of the invention, compositions comprising the polynucleotides or viral particles, and methods and uses of the polynucleotides, viral particles or compositions.

    WILSON'S DISEASE GENE THERAPY
    5.
    发明申请
    WILSON'S DISEASE GENE THERAPY 审中-公开
    WILSON's疾病基因治疗

    公开(公告)号：WO2017103624A1

    公开(公告)日：2017-06-22

    申请号：PCT/GB2016/053989

    申请日：2016-12-19

    申请人： UCL BUSINESS PLC

    发明人： NATHWANI, Amit ,  RAJ, Deepak

    IPC分类号： C12N9/14 ,  A61K48/00 ,  C12N15/864

    CPC分类号： C12N9/14 ,  A61K48/0058 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2800/22 ,  C12N2830/008

    摘要： There is described a gene therapy approach for treating Wilson's disease in which a nucleic acid molecule is used which comprises a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described are vectors comprising the nucleotide sequence and methods and uses thereof.

    摘要翻译： 描述了用于治疗威尔逊氏病的基因疗法，其中使用了包含编码功能性ATP7B蛋白的核苷酸序列的核酸分子，其中所述核苷酸序列与所述核酸分子具有至少85％的同一性 SEQ ID NO.1的序列。 还描述了包含核苷酸序列及其方法和用途的载体。

    CAPSID
    6.
    发明申请
    CAPSID 审中-公开
    衣壳

    公开(公告)号：WO2016181123A1

    公开(公告)日：2016-11-17

    申请号：PCT/GB2016/051329

    申请日：2016-05-10

    申请人： UCL BUSINESS PLC

    发明人： NATHWANI, Amit ,  DANE, Allison

    IPC分类号： A61K48/00 ,  C07K14/005 ,  C12N15/86

    CPC分类号： C07K14/005 ,  A61K48/0008 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143

    摘要： There is described an AAV capsid protein having an amino acid sequence which has at least 98% identity to the sequence of SEQ ID NO: 3 or at least 94% identity to the sequence of SEQ ID NO: 4. Also described is a pharmaceutical composition, an AAV capsid and a viral particle comprising the capsid protein, a recombinant AAV vector comprising a nucleotide sequence which encodes for the capsid protein, and a host cell and a transgenic animal comprising the capsid protein or the vector. In addition, there is described a method of transferring a nucleic acid of interest into a mammal comprising introducing a recombinant AAV vector into the mammal, wherein the recombinant AAV vector comprises a gene of interest which is encapsidated into a capsid comprising the capsid protein.

    摘要翻译： 描述了具有与SEQ ID NO：3的序列具有至少98％同一性或与SEQ ID NO：4的序列具有至少94％同一性的氨基酸序列的AAV衣壳蛋白。还描述了一种药物组合物 ，包含衣壳蛋白的AAV衣壳和病毒颗粒，包含编码衣壳蛋白的核苷酸序列的重组AAV载体，以及宿主细胞和包含衣壳蛋白或载体的转基因动物。 此外，还描述了将感兴趣的核酸转移到哺乳动物中的方法，包括将重组AAV载体导入哺乳动物，其中重组AAV载体包含被包裹在包含衣壳蛋白的衣壳中的目的基因。

    FACTOR VIII SEQUENCES
    7.
    发明申请
    FACTOR VIII SEQUENCES 审中-公开
    因子VIII序列

    公开(公告)号：WO2013186563A2

    公开(公告)日：2013-12-19

    申请号：PCT/GB2013/051551

    申请日：2013-06-12

    申请人： UCL BUSINESS PLC ,  ST JUDE CHILDREN'S RESEARCH HOSPITAL

    发明人： NATHWANI, Amit ,  MCINTOSH, Jenny ,  TUDDENHAM, Edward ,  DAVIDOFF, Andrew

    IPC分类号： C07K14/755 ,  A61K38/37

    CPC分类号： A61K38/37 ,  A01K67/0275 ,  A01K2227/105 ,  A01K2267/01 ,  A61K48/00 ,  C07K14/755

    摘要： There is provided a nucleic acid molecule comprising a nucleotide sequence encoding for a functional factor VIII protein, wherein the portion of the nucleotide sequence encoding for the B domain of the factor VIII protein is between 90 and 111 nucleotides in length and encodes for an amino acid sequence comprising a sequence having at least 85% identity to SEQ ID NO: 4 and which comprises six asparagine residues. Also provided is a functional factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating haemophilia, e.g. haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.

    摘要翻译： 提供了包含编码功能性因子VIII蛋白的核苷酸序列的核酸分子，其中编码因子VIII蛋白的B结构域的核苷酸序列的部分长度在90和111个核苷酸之间，并编码氨基酸 序列包含与SEQ ID NO：4具有至少85％同一性的序列，其包含六个天冬酰胺残基。 还提供了功能性因子VIII蛋白，包含上述核酸分子的载体，宿主细胞，转基因动物，治疗血友病的方法，例如， 血友病A，以及用于制备细小病毒基因递送载体的方法。

    ROR1 ANTIBODIES
    10.
    发明申请
    ROR1 ANTIBODIES 审中-公开

    公开(公告)号：WO2019008377A1

    公开(公告)日：2019-01-10

    申请号：PCT/GB2018/051914

    申请日：2018-07-05

    申请人： UCL BUSINESS PLC

    发明人： NATHWANI, Amit ,  GOHIL, Satyen ,  DELLA PERUTA, Marco

    IPC分类号： C07K16/28

    摘要： Thereis described Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1) antibodies that specifically bind a ROR1polypeptide, and their use. In particular, isolated monoclonal antibodies are described and their use in a number of applications, including in the detection, prevention and treatment of cancer.