AAV-based in vivo gene therapy for neurological disorders

Q Ling, JA Herstine, A Bradbury, SJ Gray - Nature Reviews Drug …, 2023 - nature.com
Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

New epilepsy therapies in development

P Klein, RM Kaminski, M Koepp… - Nature Reviews Drug …, 2024 - nature.com
Epilepsy is a common brain disorder, characterized by spontaneous recurrent seizures, with
associated neuropsychiatric and cognitive comorbidities and increased mortality. Although …

Animal models of drug-resistant epilepsy as tools for deciphering the cellular and molecular mechanisms of pharmacoresistance and discovering more effective …

W Löscher, HS White - Cells, 2023 - mdpi.com
In the last 30 years, over 20 new anti-seizure medicines (ASMs) have been introduced into
the market for the treatment of epilepsy using well-established preclinical seizure and …

Neuroimaging and multiomics reveal cross-scale circuit abnormalities in schizophrenia

M Wang, H Yan, X Tian, W Yue, Y Liu, L Fan… - Nature Mental …, 2023 - nature.com
Schizophrenia (SCZ) is a highly heterogeneous disorder with diverse clinical manifestations
and macro-and microscale biological variations, usually observed at dissociable levels …

A pH-sensitive closed-loop nanomachine to control hyperexcitability at the single neuron level

A Merolla, C Michetti, M Moschetta, F Vacca… - Nature …, 2024 - nature.com
Epilepsy affects 1% of the general population and 30% of patients are resistant to
antiepileptic drugs. Although optogenetics is an efficient antiepileptic strategy, the difficulty of …

Acetylcholine receptor based chemogenetics engineered for neuronal inhibition and seizure control assessed in mice

QA Nguyen, PM Klein, C Xie, KN Benthall… - Nature …, 2024 - nature.com
Epilepsy is a prevalent disorder involving neuronal network hyperexcitability, yet existing
therapeutic strategies often fail to provide optimal patient outcomes. Chemogenetic …

[HTML][HTML] Optogenetics for controlling seizure circuits for translational approaches

M Ledri, M Andersson, J Wickham, M Kokaia - Neurobiology of Disease, 2023 - Elsevier
The advent of optogenetic tools has had a profound impact on modern neuroscience
research, revolutionizing our understanding of the brain. These tools offer a remarkable …

Emerging molecular targets for anti-epileptogenic and epilepsy modifying drugs

K Łukasiuk, W Lasoń - International Journal of Molecular Sciences, 2023 - mdpi.com
The pharmacological treatment of epilepsy is purely symptomatic. Despite many decades of
intensive research, causal treatment of this common neurologic disorder is still unavailable …

Gene therapy for neurotransmitter‐related disorders

WS Chu, J Ng, SN Waddington… - Journal of Inherited …, 2024 - Wiley Online Library
Inborn errors of neurotransmitter (NT) metabolism are a group of rare, heterogenous
diseases with predominant neurological features, such as movement disorders, autonomic …

Reduction of Kcnt1 is therapeutic in mouse models of SCN1A and SCN8A epilepsy

SF Hill, P Jafar-Nejad, F Rigo, MH Meisler - Frontiers in Neuroscience, 2023 - frontiersin.org
Developmental and epileptic encephalopathies (DEEs) are severe seizure disorders with
inadequate treatment options. Gain-or loss-of-function mutations of neuronal ion channel …