A novel AAV9-dual microRNA-vector targeting

Mesial temporal lobe epilepsy (mTLE) is the most prevalent type of epilepsy in adults. First and subsequent generations of anti-epileptic therapy regimens fail to decrease seizures in a large number of patients suffering from mTLE, leaving surgical ablation of part of the hippocampus as the only therapeutic option to potentially reach seizure freedom. GluK2 has recently been identified as a promising target for the treatment of mTLE using gene therapy. Here, we engineered an adeno-associated virus serotype 9 vector expressing a cluster of two synthetic microRNAs (miRNAs), expressed from the human synapsin promoter, that target

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Déclaration de conflit d'intérêts

A patent application has been filed relating to this work. S.J.B., N.Pearson, C.H., N.Partouche, J.G., and R.P. declare an association with uniQure/Corlieve Therapeutics. M.W. and I.B. declare an association with uniQure B.V. A.G., J.S., A.M., and O.D. declare association with Regenxbio Inc.