VECTEURS VIRAUX DE TYPE AAV POUR LE TRAITEMENT D'UNE DYSTROPHIE RÉTINIENNE ASSOCIEE A RLBP-1

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专利权人:

Novartis AG

发明人:

申请号：

EP17204499.2

公开号：

EP3326655A1

申请日:

2013.05.02

申请国别(地区):

EP

年份:

2018

代理人:

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 Muller 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.

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