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公开(公告)号：WO2023049710A1
公开(公告)日：2023-03-30
申请号：PCT/US2022/076730
申请日：2022-09-20
IPC分类号： C07K14/005 , A61K48/00
摘要： Disclosed herein include compositions and kits comprising recombinant adeno-associated viruses (rAAVs) with tropisms to the central nervous system, the peripheral nervous system, and/or the lung, with increased specificity and transduction efficiency. Also described include methods of treating various diseases and conditions using the rAAVs.
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公开(公告)号：WO2023044306A1
公开(公告)日：2023-03-23
申请号：PCT/US2022/076363
申请日：2022-09-13
发明人： DING, Xiaozhe , GRADINARU, Viviana
IPC分类号： C07K14/005 , C12N15/86 , A61P11/00 , A61P25/00 , A61K48/00
摘要： Disclosed herein include methods and compositions comprising variant AAV capsids. Variant capsid proteins, including variant capsid proteins with structure-guided deletion/substitution, tandem multimers, and/or variant capsid proteins with structure-guided deletion and C-terminal insertion, are provided in some embodiments. The variant capsid proteins disclosed herein are capable of assembling into a variant AAV capsid with an expanded size (e.g., diameter) and/or genetic cargo capacity. Methods of treating diseases and disorders using said rAAV are also disclosed.
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公开(公告)号：WO2021183825A1
公开(公告)日：2021-09-16
申请号：PCT/US2021/022000
申请日：2021-03-11
发明人： GRADINARU, Viviana , DING, Xiaozhe
IPC分类号： C07K14/005
摘要： Disclosed herein include methods, compositions, and kits comprising variant AAV capsids. Variant capsid proteins, including guided variant capsid proteins, tandem multimers, and/or HI loop variant capsid proteins, are provided in some embodiments. The variant capsid proteins disclosed herein are capable of assembling into a variant AAV capsid with an expanded size (e.g., diameter) and/or genetic cargo capacity. Methods generating recombinant AAV (rAAV) with expanded capsids are provided. Methods of treating diseases and disorders using said rAAV are also disclosed.
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公开(公告)号：WO2017100671A1
公开(公告)日：2017-06-15
申请号：PCT/US2016/065969
申请日：2016-12-09
IPC分类号： C12N15/86 , C07K7/06 , C07K14/005
CPC分类号： C12N15/86 , A61K38/02 , A61K38/1709 , A61K38/185 , A61K38/2093 , A61K38/43 , A61K38/47 , A61K38/4813 , A61K38/50 , A61K48/00 , C07K14/005 , C07K16/18 , C07K2317/622 , C07K2319/00 , C12N7/00 , C12N15/113 , C12N15/1137 , C12N2310/122 , C12N2310/141 , C12N2320/32 , C12N2330/51 , C12N2750/14142 , C12N2750/14143 , C12N2750/14171 , C12Y302/0102 , C12Y304/14009 , C12Y305/01015 , C12N2310/14 , C12N2310/531
摘要： Disclosed herein are peptide sequences capable of directing adeno-associated viruses (AAV) to target specific environments, for example the nervous system and the heart, in a subject. Also disclosed are AAVs having non-naturally occurring capsid proteins comprising the disclosed peptide sequences, and methods of using the AAVs to treat diseases.
摘要翻译： 本文公开了能够指导腺伴随病毒（AAV）靶向特定环境（例如神经系统和心脏）的肽序列。 还公开了具有包含公开的肽序列的非天然存在的衣壳蛋白的AAV，以及使用AAV治疗疾病的方法。
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15.发明申请WHOLE-BODY TISSUE STABILIZATION AND SELECTIVE EXTRACTIONS VIA TISSUE-HYDROGEL HYBRIDS FOR HIGH RESOLUTION INTACT CIRCUIT MAPPING AND PHENOTYPING 审中-公开通过用于高分辨率电路映射和显色的组织 - 水凝胶混合物进行全身组织稳定和选择性提取
公开(公告)号：WO2017031249A1
公开(公告)日：2017-02-23
申请号：PCT/US2016/047430
申请日：2016-08-17
CPC分类号： G01N33/58 , G01N1/30 , G01N21/6458 , G01N33/50 , G01N2021/6439
摘要： In various embodiments, the present application teaches methods for tissue clearing in which tissues are rendered macromolecule-permeable and optically-transparent, thereby exposing their cellular structure with intact connectivity. In some embodiments, the present application teaches ePACT, which is a protocol for enhanced tissue clearing via expansion. In some embodiments, the present application teaches visualizing a tissue that has been expanded via ePACT.
摘要翻译： 在各种实施方案中，本申请教导了组织清除方法，其中组织被赋予大分子可渗透性和光学透明性，从而以完整的连接性暴露其细胞结构。 在一些实施方案中，本申请教导了ePACT，其是通过扩增增强组织清除的方案。 在一些实施方案中，本申请教导了通过ePACT扩增的组织的可视化。
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