Transfer of SCN1A to the brain of adolescent mouse model of Dravet syndrome improves epileptic, motor, and behavioral manifestations

iMATCH - an integrated modular assembly-system for therapeutic combination high-capacity adenovirus gene therapy

(2021) Dominik Brücher et al.   Molecular Therapy-Methods & Clinical Development

Interneuron desynchronization precedes seizures in a mouse model of Dravet syndrome

(2020) Conny H. Tran et al.   JOURNAL OF NEUROSCIENCE

Focal and generalized seizure activity after local hippocampal or cortical ablation of Na V 1.1 channels in mice

(2020) Nico A. Jansen et al.   EPILEPSIA

High-Capacity Adenoviral Vectors: Expanding the Scope of Gene Therapy

(2020) Ana Ricobaraza et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

CRISPR/dCas9-based Scn1a gene activation in inhibitory neurons ameliorates epileptic and behavioral phenotypes of Dravet syndrome model mice

(2020) Tetsushi Yamagata et al.   NEUROBIOLOGY OF DISEASE

Alterations of the Hippocampal Neurogenic Niche in a Mouse Model of Dravet Syndrome

(2020) Soraya Martín-Suárez et al.   Frontiers in Cell and Developmental Biology

A two-hit story: Seizures and genetic mutation interaction sets phenotype severity in SCN1A epilepsies

(2019) Ana Rita Salgueiro-Pereira et al.   NEUROBIOLOGY OF DISEASE

Augmented Reticular Thalamic Bursting and Seizures in Scn1a-Dravet Syndrome

(2019) Stefanie Ritter-Makinson et al.   Cell Reports

In vivo, in vitro and in silico correlations of four de novo SCN1A missense mutations

(2019) Andreea Nissenkorn et al.   PLoS One

Cerebellar, limbic, and midbrain volume alterations in sudden unexpected death in epilepsy

(2019) Luke A. Allen et al.   EPILEPSIA

SCN1A gain of function in early infantile encephalopathy

(2019) Géza Berecki et al.   ANNALS OF NEUROLOGY

The epileptic network of Lennox-Gastaut syndrome

(2019) Aaron E.L. Warren et al.   NEUROLOGY

Hippocampal deletion of NaV1.1 channels in mice causes thermal seizures and cognitive deficit characteristic of Dravet Syndrome

(2019) Rachael E. Stein et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

dCas9-based Scn1a gene activation restores inhibitory interneuron excitability and attenuates seizures in Dravet syndrome mice

(2019) Gaia Colasante et al.   MOLECULAR THERAPY

Epilepsy and neuropsychiatric comorbidities in mice carrying a recurrent Dravet syndrome SCN1A missense mutation

(2019) Ana Ricobaraza et al.   Scientific Reports

Efficient transduction of adeno-associated virus vectors into gerbil hippocampus with an appropriate combination of viral capsids and promoters

(2018) Yoshihide Sehara et al.   NEUROSCIENCE LETTERS

Listening in on the Microbubble Crowd: Advanced Acoustic Monitoring for Improved Control of Blood-Brain Barrier Opening with Focused Ultrasound

(2018) Catherine M. Gorick et al.   Theranostics

Gain of Function for the SCN1A/hNav1.1-L1670W Mutation Responsible for Familial Hemiplegic Migraine

(2018) Sandra Dhifallah et al.   Frontiers in Molecular Neuroscience

Selective NaV1.1 activation rescues Dravet syndrome mice from seizures and premature death

(2018) Kay L. Richards et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A novel GABAergic dysfunction in human Dravet syndrome

(2018) Gabriele Ruffolo et al.   EPILEPSIA

Adaptation of vectors and drug-inducible systems for controlled expression of transgenes in the tumor microenvironment

(2017) Joanna Poutou et al.   JOURNAL OF CONTROLLED RELEASE

Optimizing the Diagnosis and Management of Dravet Syndrome: Recommendations From a North American Consensus Panel

(2017) Elaine C. Wirrell et al.   PEDIATRIC NEUROLOGY

Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models

(2017) Nicola Brunetti-Pierri et al.   VIRUS GENES

Dysfunctional Brain Networking among Autonomic Regulatory Structures in Temporal Lobe Epilepsy Patients at High Risk of Sudden Unexpected Death in Epilepsy

(2017) Luke A. Allen et al.   Frontiers in Neurology

Selective spider toxins reveal a role for the Nav1.1 channel in mechanical pain

(2016) Jeremiah D. Osteen et al.   NATURE

Interictal epileptiform discharges induce hippocampal–cortical coupling in temporal lobe epilepsy

(2016) Jennifer N Gelinas et al.   NATURE MEDICINE

Upregulation of Haploinsufficient Gene Expression in the Brain by Targeting a Long Non-coding RNA Improves Seizure Phenotype in a Model of Dravet Syndrome

(2016) J. Hsiao et al.   EBioMedicine

Tumor necrosis factor-α enhances voltage-gated Na+ currents in primary culture of mouse cortical neurons

(2015) Weiqiang Chen et al.   Journal of Neuroinflammation

Sleep impairment and reduced interneuron excitability in a mouse model of Dravet Syndrome

(2015) Franck Kalume et al.   NEUROBIOLOGY OF DISEASE

Incidence of Dravet Syndrome in a US Population

(2015) Y. W. Wu et al.   PEDIATRICS

A resting-state functional connectivity study in patients at high risk for sudden unexpected death in epilepsy

(2014) Yingying Tang et al.   EPILEPSY & BEHAVIOR

Dual Transgene Expression in Murine Cerebellar Purkinje Neurons by Viral Transduction In Vivo

(2014) Marie K. Bosch et al.   PLoS One

Impaired excitability of somatostatin- and parvalbumin-expressing cortical interneurons in a mouse model of Dravet syndrome

(2014) C. Tai et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Helper Dependent Adenovirus Vectors: Progress and Future Prospects

(2013) Dan Cots et al.   CURRENT GENE THERAPY

Altered sleep regulation in a mouse model ofSCN1A-derived genetic epilepsy with febrile seizures plus (GEFS+)

(2013) Ligia A. Papale et al.   EPILEPSIA

Incidence and mechanisms of cardiorespiratory arrests in epilepsy monitoring units (MORTEMUS): a retrospective study

(2013) Philippe Ryvlin et al.   LANCET NEUROLOGY

Nonfunctional NaV1.1 familial hemiplegic migraine mutant transformed into gain of function by partial rescue of folding defects

(2013) S. Cestele et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Characterization of the Properties of Seven Promoters in the Motor Cortex of Rats and Monkeys After Lentiviral Vector-Mediated Gene Transfer

(2013) Masae Yaguchi et al.   Human Gene Therapy Methods

NaV1.1 channels are critical for intercellular communication in the suprachiasmatic nucleus and for normal circadian rhythms

(2012) S. Han et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Dravet syndrome history

(2011) CHARLOTTE DRAVET   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Molecular and cellular basis: Insights from experimental models of Dravet syndrome

(2011) Kazuhiro Yamakawa   EPILEPSIA

Retrospective multiinstitutional study of the prevalence of early death in Dravet syndrome

(2011) Masako Sakauchi et al.   EPILEPSIA

Self-inactivating helper virus for the production of high-capacity adenoviral vectors

(2011) M Gonzalez-Aparicio et al.   GENE THERAPY

Anatomical Differences Determine Distribution of Adenovirus after Convection-Enhanced Delivery to the Rat Brain

(2011) Sander Idema et al.   PLoS One

Convection-enhanced delivery and systemic mannitol increase gene product distribution of AAV vectors 5, 8, and 9 and increase gene product in the adult mouse brain

(2010) Nikisha Carty et al.   JOURNAL OF NEUROSCIENCE METHODS

Persistence of High-Capacity Adenoviral Vectors as Replication-Defective Monomeric Genomes In Vitro and in Murine Liver

(2009) Lorenz Jager et al.   HUMAN GENE THERAPY

Phenylbutyrate Ameliorates Cognitive Deficit and Reduces Tau Pathology in an Alzheimer's Disease Mouse Model

(2009) Ana Ricobaraza et al.   NEUROPSYCHOPHARMACOLOGY