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公开(公告)号：US20150087693A1
公开(公告)日：2015-03-26
申请号：US14398330
申请日：2013-05-02
申请人： The Board of Regents of the University of Texas System , The Administrators of the Tulane Educational Fund
发明人： Shusheng Wang , Eric Olson , Qinbo Zhou
IPC分类号： C12N15/113 , A61K45/06 , A61K31/713 , A61K31/7105
CPC分类号： C12N15/113 , A61K31/7105 , A61K31/713 , A61K45/06 , C12N2310/113 , C12N2310/14 , C12N2310/141 , C12N2310/3231
摘要： The present invention relates to the involvement of miR function in the development of age-related macular degeneration (AMD). It is shown that miR-23, miR-24 and/or miR-27 are involved in pathologic neovascularization in AMD, and that agonism (miR-24) and inhibition (miR23/27) of the function of these molecules blocks events contributing to development and progression of disease.
摘要翻译： 本发明涉及miR功能参与年龄相关性黄斑变性（AMD）的发展。 显示miR-23，miR-24和/或miR-27参与AMD的病理性新生血管形成，并且这些分子的功能的激动（miR-24）和抑制（miR23 / 27）阻止事件有助于 疾病的发展和进展。
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2.发明授权公开(公告)号：US10687520B2
公开(公告)日：2020-06-23
申请号：US15914728
申请日：2018-03-07
发明人： Yi-Li Min , Rhonda Bassel-Duby , Eric Olson
IPC分类号： A01K67/00 , C12N15/00 , C07H21/02 , C07H21/04 , A01K67/027 , C12N9/22 , C12N9/96 , C12N15/11 , A61K38/46 , A61K31/7105 , A61P21/00 , C12N15/113 , A61K48/00 , C12N15/85
摘要： Duchenne muscular dystrophy (DMD), which affects 1 in 5,000 male births, is one of the most common genetic disorders of children. This disease is caused by an absence or deficiency of dystrophin protein in striated muscle. The major DMD deletion “hot spots” are found between exon 6 to 8, and exons 45 to 53. Here, a “humanized” mouse model is provided that can be used to test a variety of DMD exon skipping strategies. Among these are, CRISPR/Cas9 oligonucleotides, small molecules or other therapeutic modalities that promote exon skipping or micro dystrophin mini genes or cell based therapies. Methods for restoring the reading frame of exon 44 deletion via CRISPR-mediated exon skipping in the humanized mouse model, in patient-derived iPS cells and ultimately, in patients using various delivery systems are also contemplated. The impact of CRISPR technology on DMD is that gene editing can permanently correct mutations.
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公开(公告)号：US09598694B2
公开(公告)日：2017-03-21
申请号：US14398330
申请日：2013-05-02
发明人： Shusheng Wang , Eric Olson , Qinbo Zhou
IPC分类号： C12N15/113 , A61K31/7105 , A61K31/713 , A61K45/06
CPC分类号： C12N15/113 , A61K31/7105 , A61K31/713 , A61K45/06 , C12N2310/113 , C12N2310/14 , C12N2310/141 , C12N2310/3231
摘要： The present invention relates to the involvement of miR function in the development of age-related macular degeneration (AMD). It is shown that miR-23, miR-24 and/or miR-27 are involved in pathologic neovascularization in AMD, and that agonism (miR-24) and inhibition (miR23/27) of the function of these molecules blocks events contributing to development and progression of disease.
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公开(公告)号：US11471509B2
公开(公告)日：2022-10-18
申请号：US16379610
申请日：2019-04-09
发明人： Eric Olson , Douglas P. Millay
摘要： The present disclosure describes the fusogenic activity of the Myomaker protein. This polypeptide, when expressed in non-muscle cells, is able to drive fusion of the cell with a muscle cell, but not with other non-muscle cells. The use of this protein and cell expressing it in the delivery of exogenous genetic material to muscle cells also is described.
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公开(公告)号：US10272137B2
公开(公告)日：2019-04-30
申请号：US14900005
申请日：2014-06-27
发明人： Eric Olson , Douglas P. Millay
摘要： The present disclosure describes the fusogenic activity of the Myomaker protein. This polypeptide, when expressed in non-muscle cells, is able to drive fusion of the cell with a muscle cell, but not with other non-muscle cells. The use of this protein and cell expressing it in the delivery of exogenous genetic material to muscle cells also is described.
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