Post-modern therapeutic approaches for progressive myoclonus epilepsy

Research output: Contribution to journalReview article

3 Citations (Scopus)

Abstract

While the PME are arguably the severest epilepsies and neurological disorders, the vast majority are monogenic. Additionally, many affect straightforward biochemical pathways. Finally, by definition, they occur in previously healthy and well-developed brains. As such, their therapies should be easier than in complex, albeit often less severe, neurological developmental disorders where the complex, poorly understood, and extremely difficult-to-correct, neural network of the brain is affected. This last article reviews the latest cutting edge technologies in monogenic disease therapy, with some examples provided applicable to a number of disease. It aims to give a sense of where we are and how much closer we are, to the goal of making an actual organic difference.

Original languageEnglish (US)
Pages (from-to)S154-S158
JournalEpileptic Disorders
Volume18
DOIs
StatePublished - Jan 1 2016

Fingerprint

Progressive Myoclonic Epilepsy
Nervous System Diseases
Brain
Epilepsy
Technology
Therapeutics

Keywords

  • AAV9
  • Cas9
  • CRISPR
  • gene therapy
  • progressive myoclonus epilepsies
  • small molecule

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

Cite this

Post-modern therapeutic approaches for progressive myoclonus epilepsy. / Minassian, Berge A.

In: Epileptic Disorders, Vol. 18, 01.01.2016, p. S154-S158.

Research output: Contribution to journalReview article

@article{c832f669e76c4938a59eb97769251494,
title = "Post-modern therapeutic approaches for progressive myoclonus epilepsy",
abstract = "While the PME are arguably the severest epilepsies and neurological disorders, the vast majority are monogenic. Additionally, many affect straightforward biochemical pathways. Finally, by definition, they occur in previously healthy and well-developed brains. As such, their therapies should be easier than in complex, albeit often less severe, neurological developmental disorders where the complex, poorly understood, and extremely difficult-to-correct, neural network of the brain is affected. This last article reviews the latest cutting edge technologies in monogenic disease therapy, with some examples provided applicable to a number of disease. It aims to give a sense of where we are and how much closer we are, to the goal of making an actual organic difference.",
keywords = "AAV9, Cas9, CRISPR, gene therapy, progressive myoclonus epilepsies, small molecule",
author = "Minassian, {Berge A.}",
year = "2016",
month = "1",
day = "1",
doi = "10.1684/epd.2016.0862",
language = "English (US)",
volume = "18",
pages = "S154--S158",
journal = "Epileptic Disorders",
issn = "1294-9361",
publisher = "Springer Paris",

}

TY - JOUR

T1 - Post-modern therapeutic approaches for progressive myoclonus epilepsy

AU - Minassian, Berge A.

PY - 2016/1/1

Y1 - 2016/1/1

N2 - While the PME are arguably the severest epilepsies and neurological disorders, the vast majority are monogenic. Additionally, many affect straightforward biochemical pathways. Finally, by definition, they occur in previously healthy and well-developed brains. As such, their therapies should be easier than in complex, albeit often less severe, neurological developmental disorders where the complex, poorly understood, and extremely difficult-to-correct, neural network of the brain is affected. This last article reviews the latest cutting edge technologies in monogenic disease therapy, with some examples provided applicable to a number of disease. It aims to give a sense of where we are and how much closer we are, to the goal of making an actual organic difference.

AB - While the PME are arguably the severest epilepsies and neurological disorders, the vast majority are monogenic. Additionally, many affect straightforward biochemical pathways. Finally, by definition, they occur in previously healthy and well-developed brains. As such, their therapies should be easier than in complex, albeit often less severe, neurological developmental disorders where the complex, poorly understood, and extremely difficult-to-correct, neural network of the brain is affected. This last article reviews the latest cutting edge technologies in monogenic disease therapy, with some examples provided applicable to a number of disease. It aims to give a sense of where we are and how much closer we are, to the goal of making an actual organic difference.

KW - AAV9

KW - Cas9

KW - CRISPR

KW - gene therapy

KW - progressive myoclonus epilepsies

KW - small molecule

UR - http://www.scopus.com/inward/record.url?scp=84994108272&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84994108272&partnerID=8YFLogxK

U2 - 10.1684/epd.2016.0862

DO - 10.1684/epd.2016.0862

M3 - Review article

VL - 18

SP - S154-S158

JO - Epileptic Disorders

JF - Epileptic Disorders

SN - 1294-9361

ER -