On-demand cell-autonomous gene therapy for brain circuit disorders

Y Qiu, N O'Neill, B Maffei, C Zourray… - Science, 2022 - science.org
Several neurodevelopmental and neuropsychiatric disorders are characterized by
intermittent episodes of pathological activity. Although genetic therapies offer the ability to …

Biochemical autoregulatory gene therapy for focal epilepsy

A Lieb, Y Qiu, CL Dixon, JP Heller, MC Walker… - Nature medicine, 2018 - nature.com
Despite the introduction of more than one dozen new antiepileptic drugs in the past 20
years, approximately one-third of people who develop epilepsy continue to have seizures on …

Inducible genetic suppression of neuronal excitability

DC Johns, R Marx, RE Mains, B O'Rourke… - Journal of …, 1999 - Soc Neuroscience
Graded, reversible suppression of neuronal excitability represents a logical goal of therapy
for epilepsy and intractable pain. To achieve such suppression, we have developed the …

Emerging insights into the genesis of epilepsy

JO McNamara - Nature, 1999 - nature.com
Epilepsies are a diverse collection of brain disorders that affect 1–2% of the population.
Current therapies are unsatisfactory as they provide only symptomatic relief, are effective in …

Epilepsy gene therapy using an engineered potassium channel

A Snowball, E Chabrol, RC Wykes… - Journal of …, 2019 - Soc Neuroscience
Refractory focal epilepsy is a devastating disease for which there is frequently no effective
treatment. Gene therapy represents a promising alternative, but treating epilepsy in this way …

Pathway-driven discovery of epilepsy genes

J Noebels - Nature neuroscience, 2015 - nature.com
Epilepsy genes deliver critical insights into the molecular control of brain synchronization
and are revolutionizing our understanding and treatment of the disease. The epilepsy …

Transient cortical circuits match spontaneous and sensory-driven activity during development

Z Molnár, HJ Luhmann, PO Kanold - Science, 2020 - science.org
BACKGROUND During early mammalian brain development, transient neurons and circuits
form the scaffold for the development of neuronal networks. In the immature cerebral cortex …

Optogenetic and potassium channel gene therapy in a rodent model of focal neocortical epilepsy

RC Wykes, JH Heeroma, L Mantoan, K Zheng… - Science translational …, 2012 - science.org
Neocortical epilepsy is frequently drug-resistant. Surgery to remove the epileptogenic zone
is only feasible in a minority of cases, leaving many patients without an effective treatment …

Targeting epilepsy genes

JL Noebels - Neuron, 1996 - cell.com
Jeffrey L. Noebels steps taken by gene errors to destabilize neural net-Developmental
Neurogenetics Laboratory works; the first practical issue is which gene to select. Department …

Treatment during a vulnerable developmental period rescues a genetic epilepsy

SL Marguet, VTQ Le-Schulte, A Merseburg, A Neu… - Nature medicine, 2015 - nature.com
The nervous system is vulnerable to perturbations during specific developmental periods.
Insults during such susceptible time windows can have long-term consequences, including …