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 …
treatment of neurological disorders. Among the available delivery vehicles, adeno …
New epilepsy therapies in development
Epilepsy is a common brain disorder, characterized by spontaneous recurrent seizures, with
associated neuropsychiatric and cognitive comorbidities and increased mortality. Although …
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 …
the market for the treatment of epilepsy using well-established preclinical seizure and …
Neuroimaging and multiomics reveal cross-scale circuit abnormalities in schizophrenia
Schizophrenia (SCZ) is a highly heterogeneous disorder with diverse clinical manifestations
and macro-and microscale biological variations, usually observed at dissociable levels …
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 …
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
Epilepsy is a prevalent disorder involving neuronal network hyperexcitability, yet existing
therapeutic strategies often fail to provide optimal patient outcomes. Chemogenetic …
therapeutic strategies often fail to provide optimal patient outcomes. Chemogenetic …
[HTML][HTML] Optogenetics for controlling seizure circuits for translational approaches
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 …
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 …
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 …
diseases with predominant neurological features, such as movement disorders, autonomic …
Reduction of Kcnt1 is therapeutic in mouse models of SCN1A and SCN8A epilepsy
Developmental and epileptic encephalopathies (DEEs) are severe seizure disorders with
inadequate treatment options. Gain-or loss-of-function mutations of neuronal ion channel …
inadequate treatment options. Gain-or loss-of-function mutations of neuronal ion channel …