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公开(公告)号：US09163259B2
公开(公告)日：2015-10-20
申请号：US13873558
申请日：2013-04-30
CPC分类号： C12N15/86 , A61K38/1709 , A61K48/005 , A61K48/0058 , C07H21/04 , C07K14/47 , C12N7/00 , C12N15/79 , C12N2750/14143 , C12N2750/14171 , C12N2750/14343 , C12N2750/14345 , C12N2750/14371 , C12N2800/22 , C12N2810/6027 , C12N2810/85
摘要： The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.
摘要翻译： 本发明涉及能够将异源基因递送至视网膜，特别是将RLBP1递送至视网膜的RPE和Müller细胞的病毒载体。 本发明还涉及可用于产生病毒载体的核酸，包含病毒载体的组合物和组合物和病毒载体的用途。 本发明还涉及向视网膜递送和/或表达异源基因的方法，改善受试者中暗适应的速率并治疗RLBP1相关的视网膜营养不良症。
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公开(公告)号：US20160097061A1
公开(公告)日：2016-04-07
申请号：US14881960
申请日：2015-10-13
CPC分类号： C12N15/86 , A61K38/1709 , A61K48/005 , A61K48/0058 , C07H21/04 , C07K14/47 , C12N7/00 , C12N15/79 , C12N2750/14143 , C12N2750/14171 , C12N2750/14343 , C12N2750/14345 , C12N2750/14371 , C12N2800/22 , C12N2810/6027 , C12N2810/85
摘要： The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.
摘要翻译： 本发明涉及能够将异源基因递送至视网膜，特别是将RLBP1递送至视网膜的RPE和Müller细胞的病毒载体。 本发明还涉及可用于产生病毒载体的核酸，包含病毒载体的组合物和组合物和病毒载体的用途。 本发明还涉及向视网膜递送和/或表达异源基因的方法，改善受试者中暗适应的速率并治疗RLBP1相关的视网膜营养不良症。
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公开(公告)号：US20170290876A1
公开(公告)日：2017-10-12
申请号：US15315626
申请日：2015-06-24
申请人： Joy GHOSH , Michael ROGUSKA , Andrew Anh NGUYEN , Thomas PIETZONKA , Stephen Hedrick POOR , Matthias MACHACEK , Chad Eric BIGELOW
发明人： Joy GHOSH , Michael ROGUSKA , Andrew Anh NGUYEN , Thomas PIETZONKA , Stephen Hedrick POOR , Matthias MACHACEK , Chad Eric BIGELOW
IPC分类号： A61K38/00 , A61K31/7105 , A61K31/711 , A61K38/02 , A61K45/06
CPC分类号： A61K38/005 , A61K31/7105 , A61K31/711 , A61K38/02 , A61K45/06 , A61K2121/00 , C07K2319/31 , C12N15/62
摘要： The present invention is based, in part, on the discovery of peptide tags that increase the half-life and/or mean residence time of proteins or nucleic acids in the eye. In certain aspects the invention peptide tags increase the half-life and/or mean residence time of antibodies and antigen binding fragments, therapeutic proteins, protein receptors, DARPins and/or aptamers in the eye.
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公开(公告)号：US20140017201A1
公开(公告)日：2014-01-16
申请号：US13873558
申请日：2013-04-30
CPC分类号： C12N15/86 , A61K38/1709 , A61K48/005 , A61K48/0058 , C07H21/04 , C07K14/47 , C12N7/00 , C12N15/79 , C12N2750/14143 , C12N2750/14171 , C12N2750/14343 , C12N2750/14345 , C12N2750/14371 , C12N2800/22 , C12N2810/6027 , C12N2810/85
摘要： The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.
摘要翻译： 本发明涉及能够将异源基因递送至视网膜，特别是将RLBP1递送至视网膜的RPE和Müller细胞的病毒载体。 本发明还涉及可用于产生病毒载体的核酸，包含病毒载体的组合物和组合物和病毒载体的用途。 本发明还涉及向视网膜递送和/或表达异源基因的方法，改善受试者中暗适应的速率并治疗RLBP1相关的视网膜营养不良症。
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