发明公开
EP2844302A2 VIRAL VECTORS FOR THE TREATMENT OF RETINAL DYSTROPHY 审中-公开
VIRALE VEKTOREN ZUR BEHANDLUNG VON RETINALER DYSTROPHIE

VIRAL VECTORS FOR THE TREATMENT OF RETINAL DYSTROPHY
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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