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公开(公告)号：EP3736330A1
公开(公告)日：2020-11-11
申请号：EP19173365.8
申请日：2019-05-08
摘要： The present invention relates to improved adeno-associated virus (AAV) particles for gene delivery and gene therapy. Provided are adeno associated virus (AAV) particles that comprise a modified capsid. The present invention further relates to methods for producing the improved AAV particles of this invention by removing natural binding sites in adeno associated virus (AAV) capsids and introducing ligand binding sites into said capsid to provide AAVs that transduce only particular cells of interest. An additional aspect of the present invention relates to modified AAV particles for use in the treatment of a disease and methods for treating a disease, comprising administering the modified AAV particles to a subject in need thereof. Yet a further aspect of this invention relates to the AAV particles of this invention for the transfection of cells, for example as a gene delivery tool in basic research.
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公开(公告)号：EP3966319A2
公开(公告)日：2022-03-16
申请号：EP20723864.3
申请日：2020-05-07
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公开(公告)号：EP3506928A2
公开(公告)日：2019-07-10
申请号：EP17765380.5
申请日：2017-09-01
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公开(公告)号：EP3402325A1
公开(公告)日：2018-11-21
申请号：EP16822208.1
申请日：2016-12-28
发明人： HEPPENSTALL, Paul , MORLEY, Shane
IPC分类号： A01K67/027
CPC分类号： A01K67/0276 , A01K2217/075 , A01K2217/206 , A01K2227/105 , A01K2267/0356
摘要： The present invention pertains to novel analgesics useful for treating mechanical pain. The invention suggests the use of inhibitors of α-tubulin acetylation for inhibition of neurological sensations that are mediated by sensory neurons. The perception of mechanical pain is can be modulated by altering the α-tubulin acetylation, in context of the invention in particular by modulation of the expression and/or activity of the enzyme α-tubulin acetyltransferase (Atat). The invention provides the medical application of α-tubulin acetyltransferase inhibitors as analgesics and a screening method for the identification of compounds useful in the treatment of pain.
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