Barriers to the use of genetic information for the development of new epilepsy treatments

Research output: Contribution to journalEditorial

1 Citation (Scopus)

Abstract

Genetic analysis is providing new information on the biological basis of epilepsy at a rapid pace; this article identifies factors acting as major barriers to use of these data for therapy development. Disease heterogeneity is a primary obstacle since so many genes can cause or predispose to epilepsy and the clinical presentation of epilepsy is so diverse, thus making it difficult to define the most therapeutically relevant targets. Further, many epilepsy genes affect brain development, an observation that represents a barrier unto itself given the challenge of reversing or preventing genetically mediated alterations of brain pathway formation. Finally, the lack of appropriate models for testing new therapies is also recognized as a fundamental limitation. Overcoming these barriers will be aided by full characterization of the genetic landscape of epilepsy, elucidation of key pathway points for therapeutic intervention and creation of unique experimental models to validate results.

Original languageEnglish (US)
Pages (from-to)5-8
Number of pages4
JournalExpert Review of Neurotherapeutics
Volume16
Issue number1
DOIs
StatePublished - Jan 2 2016

Fingerprint

Epilepsy
Brain
Genes
Theoretical Models
Therapeutics
Observation

All Science Journal Classification (ASJC) codes

  • Neuroscience(all)
  • Clinical Neurology
  • Pharmacology (medical)

Cite this

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abstract = "Genetic analysis is providing new information on the biological basis of epilepsy at a rapid pace; this article identifies factors acting as major barriers to use of these data for therapy development. Disease heterogeneity is a primary obstacle since so many genes can cause or predispose to epilepsy and the clinical presentation of epilepsy is so diverse, thus making it difficult to define the most therapeutically relevant targets. Further, many epilepsy genes affect brain development, an observation that represents a barrier unto itself given the challenge of reversing or preventing genetically mediated alterations of brain pathway formation. Finally, the lack of appropriate models for testing new therapies is also recognized as a fundamental limitation. Overcoming these barriers will be aided by full characterization of the genetic landscape of epilepsy, elucidation of key pathway points for therapeutic intervention and creation of unique experimental models to validate results.",
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Barriers to the use of genetic information for the development of new epilepsy treatments. / Ferraro, Thomas N.

In: Expert Review of Neurotherapeutics, Vol. 16, No. 1, 02.01.2016, p. 5-8.

Research output: Contribution to journalEditorial

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