TY - JOUR
AU1 - Lieb, Andreas
AU2 - Qiu, Yichen
AU3 - Dixon, Christine L
AU4 - Heller, Janosch P
AU5 - Walker, Matthew C
AU6 - Schorge, Stephanie
AU7 - Kullmann, Dimitri M
AB - Despite the introduction of over a dozen new antiepileptic drugs in the last
20 years, approximately one third of people who develop epilepsy continue to have
seizures on mono- or polytherapy1.
Viral-vector mediated gene transfer offers the opportunity to design a rational
treatment that builds on mechanistic understanding of seizure generation and that
can be targeted to specific neuronal populations in epileptogenic foci2. Several such strategies show encouraging
results in different animal models, although clinical translation is limited by
possible effects on circuits underlying cognitive, mnemonic, sensory or motor
function. Here we describe an autoregulatory anti-epileptic gene therapy, which
relies on neuronal inhibition in response to elevations of extracellular glutamate.
It is effective in a rodent model of focal epilepsy, and well tolerated, thus
lowering the barrier to clinical translation.
TI - Biochemical autoregulatory gene therapy for focalepilepsy
JF - Nature Medicine
DO - 10.1038/s41591-018-0103-x
DA - 2018-07-09
UR - https://www.deepdyve.com/lp/pubmed-central/biochemical-autoregulatory-gene-therapy-for-focalepilepsy-IQWQfs0TG7
SP - 1324
EP - 1329
VL - 24
IS - 9
DP - DeepDyve
ER -