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WONOEP appraisal: Targeted therapy development for early onset epilepsies.

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Title: WONOEP appraisal: Targeted therapy development for early onset epilepsies.
Authors: Casillas‐Espinosa, Pablo M. (AUTHOR), Wong, Jennifer C. (AUTHOR), Grabon, Wanda (AUTHOR), Gonzalez‐Ramos, Ana (AUTHOR), Mantegazza, Massimo (AUTHOR), Yilmaz, Nihan Carcak (AUTHOR), Patel, Manisha (AUTHOR), Staley, Kevin (AUTHOR), Sankar, Raman (AUTHOR), O'Brien, Terence J. (AUTHOR), Akman, Özlem (AUTHOR), Balagura, Ganna (AUTHOR), Numis, Adam L. (AUTHOR), Noebels, Jeffrey L. (AUTHOR), Baulac, Stéphanie (AUTHOR), Auvin, Stéphane (AUTHOR), Henshall, David C. (AUTHOR), Galanopoulou, Aristea S. (AUTHOR)
Source: Epilepsia (Series 4). Feb2025, Vol. 66 Issue 2, p328-340. 13p.
Subjects: Seizures (Medicine), Childhood epilepsy, Cannabinoid receptors, Heredity, Chloride channels, Gene therapy, Individualized medicine
Abstract: The early onset epilepsies encompass a heterogeneous group of disorders, some of which result in drug‐resistant seizures, developmental delay, psychiatric comorbidities, and sudden death. Advancement in the widespread use of targeted gene panels as well as genome and exome sequencing has facilitated the identification of different causative genes in a subset of these patients. The ability to recognize the genetic basis of early onset epilepsies continues to improve, with de novo coding variants accounting for most of the genetic etiologies identified. Although current disease‐specific and disease‐modifying therapies remain limited, novel precision medicine approaches, such as small molecules, cell therapy, and other forms of genetic therapies for early onset epilepsies, have created excitement among researchers, clinicians, and caregivers. Here, we summarize the main findings of presentations and discussions on novel therapeutic strategies for targeted treatment of early onset epilepsies that occurred during the Workshop on Neurobiology of Epilepsy (WONOEP XVI, Talloires, France, July 2022). The presentations discussed the use of chloride transporter inhibitors for neonatal seizures, targeting orexinergic signaling for childhood absence epilepsy, targeting energy metabolism in Dravet syndrome, and the role of cannabinoid receptor type 2, reversible acetylcholinesterase inhibitors, cell therapies, and RNA‐based therapies in early life epilepsies. [ABSTRACT FROM AUTHOR]
Copyright of Epilepsia (Series 4) is the property of Wiley-Blackwell and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
Database: Psychology and Behavioral Sciences Collection
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PubType: Academic Journal
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  Data: WONOEP appraisal: Targeted therapy development for early onset epilepsies.
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  Data: <searchLink fieldCode="AR" term="%22Casillas‐Espinosa%2C+Pablo+M%2E%22">Casillas‐Espinosa, Pablo M.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Wong%2C+Jennifer+C%2E%22">Wong, Jennifer C.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Grabon%2C+Wanda%22">Grabon, Wanda</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Gonzalez‐Ramos%2C+Ana%22">Gonzalez‐Ramos, Ana</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Mantegazza%2C+Massimo%22">Mantegazza, Massimo</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Yilmaz%2C+Nihan+Carcak%22">Yilmaz, Nihan Carcak</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Patel%2C+Manisha%22">Patel, Manisha</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Staley%2C+Kevin%22">Staley, Kevin</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Sankar%2C+Raman%22">Sankar, Raman</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22O'Brien%2C+Terence+J%2E%22">O'Brien, Terence J.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Akman%2C+Özlem%22">Akman, Özlem</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Balagura%2C+Ganna%22">Balagura, Ganna</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Numis%2C+Adam+L%2E%22">Numis, Adam L.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Noebels%2C+Jeffrey+L%2E%22">Noebels, Jeffrey L.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Baulac%2C+Stéphanie%22">Baulac, Stéphanie</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Auvin%2C+Stéphane%22">Auvin, Stéphane</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Henshall%2C+David+C%2E%22">Henshall, David C.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Galanopoulou%2C+Aristea+S%2E%22">Galanopoulou, Aristea S.</searchLink> (AUTHOR)
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  Data: <searchLink fieldCode="JN" term="%22Epilepsia+%28Series+4%29%22">Epilepsia (Series 4)</searchLink>. Feb2025, Vol. 66 Issue 2, p328-340. 13p.
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  Data: The early onset epilepsies encompass a heterogeneous group of disorders, some of which result in drug‐resistant seizures, developmental delay, psychiatric comorbidities, and sudden death. Advancement in the widespread use of targeted gene panels as well as genome and exome sequencing has facilitated the identification of different causative genes in a subset of these patients. The ability to recognize the genetic basis of early onset epilepsies continues to improve, with de novo coding variants accounting for most of the genetic etiologies identified. Although current disease‐specific and disease‐modifying therapies remain limited, novel precision medicine approaches, such as small molecules, cell therapy, and other forms of genetic therapies for early onset epilepsies, have created excitement among researchers, clinicians, and caregivers. Here, we summarize the main findings of presentations and discussions on novel therapeutic strategies for targeted treatment of early onset epilepsies that occurred during the Workshop on Neurobiology of Epilepsy (WONOEP XVI, Talloires, France, July 2022). The presentations discussed the use of chloride transporter inhibitors for neonatal seizures, targeting orexinergic signaling for childhood absence epilepsy, targeting energy metabolism in Dravet syndrome, and the role of cannabinoid receptor type 2, reversible acetylcholinesterase inhibitors, cell therapies, and RNA‐based therapies in early life epilepsies. [ABSTRACT FROM AUTHOR]
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  Data: <i>Copyright of Epilepsia (Series 4) is the property of Wiley-Blackwell and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
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        Value: 10.1111/epi.18187
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        Text: English
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