Recent Patents on DNA & Gene Sequences 2009, 3, 183-192 183
1872-2156/09 $100.00+.00 © 2009 Bentham Science Publishers Ltd.
Recent Patents on Epilepsy Genetics
Valerio Napolioni*
Laboratory of Human Genetics, Department of Molecular, Cellular and Animal Biology, University of Camerino,
Camerino, Italy
Received: July 23, 2009; Accepted: August 11, 2009; Revised: August 17, 2009
Abstract: The field of epilepsy genetics is contentious, particularly when it concerns the common epilepsies. More than a
dozen loci have been suggested, the result of either linkage analysis and/or association analysis, but few of these findings
have been replicated, let alone proven, and those that have are mostly for rare forms of epilepsy. Molecular genetics has
revolutionised the understanding of epilepsy genetics and is beginning to have a significant clinical impact. Technological
advances have resulted in new high-throughput approaches that promise to further our understanding of the molecular
genetics of the epilepsies.
The patents discussed in this review highlight the important discoveries that have contributed to our understanding of
epilepsy genetics and provide valuable information as to where research in this area will be heading in the future. This
knowledge not only informs clinicians about the biology of the epilepsies but also has important consequences for clinical
practice and genetic counselling.
Keywords: Epilepsy, idiopathic generalized epilepsy, channelopathies, monogenic disease, complex phenotype, mutations,
single nucleotide polymorphism.
1. INTRODUCTION
Epilepsy is a chronic neurological condition that involves
seizure recurrence. A seizure occurs when abnormal
electrical activity in the brain causes an involuntary change
in body movement or function, sensation, awareness, or
behavior.
Seizures can vary from a momentary disruption of the
senses, to short periods of unconsciousness or staring spells,
to convulsions. The disease is characterised by neuronal
hyper-excitability, and is associated with mutations in
several genes involved in nerve communication or energy
production. Epilepsy can occur also as a result of many
different conditions that affect a person’s brain. Examples of
these conditions include stroke, complications during
childbirth, infections (such as meningitis, encephalitis,
cysticercosis, or brain abscess), head trauma, and certain
genetic disorders. Often, no definite cause can be found.
Approximately 9% of the population have a seizure at
some stage in their lifetime and 3% develop epilepsy. The
World Health Organization estimates that epilepsy affects
approximately 50 million people worldwide, with approxi-
mately 2.5 million cases in the US.
Epilepsy should not be understood as a single disorder,
but rather as a group of syndromes with vastly divergent
symptoms but all involving episodic abnormal electrical
activity in the brain.
*Address correspondence to this author at the Laboratory of Human
Genetics, Department of Molecular, Cellular and Animal Biology,
University of Camerino, Camerino, Italy; Tel: 39-0737403293; Fax: 39-
0737403290; E-mail: valerio.napolioni@unicam.it
In 1981, the International League Against Epilepsy
(ILAE) proposed a classification scheme for individual
seizures that remains in common use [1]. This classification
is based on observation (clinical and EEG) rather than the
underlying pathophysiology or anatomy and is outlined later
on in this article. In 1989, the ILAE proposed a classification
scheme for epilepsies and epileptic syndromes [2].
Genetic factors are now recognised to have an even more
important role in epilepsies than previously appreciated.
Rare mendelian forms of epilepsy are now well recog-
nised, and there is evidence of complex inheritance due to
multiple susceptibility genes in most idiopathic epilepsies.
The complexities of epilepsy classification and the variety of
clinical genetic methodologies (family aggregation, twin,
and multiplex family studies) have led to an apparently
confusing picture. Molecular approaches have revealed
genes for many mendelian epilepsies Table 1. Most encode
ion channel subunits, but major challenges remain in
understanding phenotype–genotype relationships. These
challenges are even greater in complex epilepsies in which
gene discovery is still in its infancy.
The present article reviews a number of recently issued
patents and recently published patent applications that have
identified new epilepsy genes and uses thereof. The
identification of these “epilepsy-genes” will open the road to
molecular diagnostics and will provide clinicians an
important tool for treatment design.
2. IDIOPATHIC EPILEPSIES
Idiopathic epilepsies have been divided into partial and
generalized sub-types Table 1. Partial (focal or local)
epileptic fits arise from localized cortical discarges, so that