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公开(公告)号：US07041448B2
公开(公告)日：2006-05-09
申请号：US09851494
申请日：2001-05-08
CPC分类号： C07K14/705 , C12Q1/6883 , C12Q2600/156 , C12Q2600/158 , C12Q2600/172
摘要： The present invention relates to identification of a gene that is inactivated in a mucolipidosis condition. In particular, the invention concerns mutations that disrupt a mucolipin, preferably MCOLN1, in mucolipidosis IV. Recombinant nucleic acids encoding mutant forms of MCOLN1, oligonucleotides specific for such mutations, and diagnostic and therapeutic applications related to these discoveries, are also contemplated.
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2.发明申请Methods for Altering MRNA Splicing and Treating Familial Dysautonomia and Other Mechanistically Related Disorders 有权改变MRNA切割和治疗家族性自主神经病和其他机械相关性疾病的方法公开(公告)号：US20120329816A1
公开(公告)日：2012-12-27
申请号：US13601034
申请日：2012-08-31
CPC分类号： A61K31/52 , A61K31/353 , A61K31/355 , C12Q1/6883 , C12Q2600/136 , C12Q2600/156 , C12Q2600/158 , A61K2300/00
摘要： This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
摘要翻译： 本发明涉及改变细胞中mRNA的剪接的方法。 特别地，本发明还涉及用于增加具有编码与突变蛋白相对应的i）错过的mRNA的突变基因的细胞中野生型与错合型mRNA和相应编码蛋白的比例的方法，或ii） 拼接机构负责处理错误的mRNA。 此外，本发明涉及通过向这样的个体施用诸如激动素的细胞分裂素来治疗与错过的mRNA相关的病症的个体，例如家族性自主神经病或神经纤维瘤病1。
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3.发明授权Methods for altering MRNA splicing and treating familial dysautonomia by administering kinetin, benzyladenine, and tocotrienols 有权通过施用激动素，苄基腺嘌呤和生育三烯酚来改变MRNA剪接和治疗家族性不自主的方法公开(公告)号：US08729025B2
公开(公告)日：2014-05-20
申请号：US13601034
申请日：2012-08-31
CPC分类号： A61K31/52 , A61K31/353 , A61K31/355 , C12Q1/6883 , C12Q2600/136 , C12Q2600/156 , C12Q2600/158 , A61K2300/00
摘要： This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
摘要翻译： 本发明涉及改变细胞中mRNA的剪接的方法。 特别地，本发明还涉及用于增加具有编码与突变蛋白相对应的i）错过的mRNA的突变基因的细胞中野生型与错合型mRNA和相应编码蛋白的比例的方法，或ii） 拼接机构负责处理错误的mRNA。 此外，本发明涉及通过向这样的个体施用诸如激动素的细胞分裂素来治疗与错过的mRNA相关的病症的个体，例如家族性自主神经病或神经纤维瘤病1。
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4.发明申请Methods for Altering MRNA Splicing and Treating Familial Dysautonomia and Other Mechanistically Related Disorders 审中-公开改变MRNA切割和治疗家族性自主神经病和其他机械相关性疾病的方法公开(公告)号：US20110136836A1
公开(公告)日：2011-06-09
申请号：US12773137
申请日：2010-05-04
CPC分类号： A61K31/52 , A61K31/353 , A61K31/355 , C12Q1/6883 , C12Q2600/136 , C12Q2600/156 , C12Q2600/158 , A61K2300/00
摘要： This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
摘要翻译： 本发明涉及改变细胞中mRNA的剪接的方法。 特别地，本发明还涉及用于增加具有编码与突变蛋白相对应的i）错过的mRNA的突变基因的细胞中野生型与错合型mRNA和相应编码蛋白的比例的方法，或ii） 拼接机构负责处理错误的mRNA。 此外，本发明涉及通过向这样的个体施用诸如激动素的细胞分裂素来治疗与错过的mRNA相关的病症的个体，例如家族性自主神经病或神经纤维瘤病1。
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5.发明授权Methods for altering mRNA splicing and treating familial dysautonomia and other mechanistically related disorders 有权改变mRNA剪接和治疗家族性肥胖症等机械相关疾病的方法公开(公告)号：US07737110B2
公开(公告)日：2010-06-15
申请号：US10956601
申请日：2004-10-01
CPC分类号： A61K31/52 , A61K31/353 , A61K31/355 , C12Q1/6883 , C12Q2600/136 , C12Q2600/156 , C12Q2600/158 , A61K2300/00
摘要： This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
摘要翻译： 本发明涉及改变细胞中mRNA的剪接的方法。 特别地，本发明还涉及用于增加具有编码与突变蛋白相对应的i）错过的mRNA的突变基因的细胞中野生型与错合型mRNA和相应编码蛋白的比例的方法，或者ii） 拼接机构负责处理错误的mRNA。 此外，本发明涉及通过向这样的个体施用诸如激动素的细胞分裂素来治疗与错过的mRNA相关的病症的个体，例如家族性自主神经病或神经纤维瘤病1。
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