The neurobiological basis for novel experimental therapeutics in dystonia

Anthony M. Downs, Kaitlyn M. Roman, Simone A. Campbell, Antonio Pisani, Ellen J. Hess, P. Bonsi

Research output: Contribution to journalReview article

Abstract

Dystonia is a movement disorder characterized by involuntary muscle contractions, twisting movements, and abnormal postures that may affect one or multiple body regions. Dystonia is the third most common movement disorder after Parkinson's disease and essential tremor. Despite its relative frequency, small molecule therapeutics for dystonia are limited. Development of new therapeutics is further hampered by the heterogeneity of both clinical symptoms and etiologies in dystonia. Recent advances in both animal and cell-based models have helped clarify divergent etiologies in dystonia and have facilitated the identification of new therapeutic targets. Advances in medicinal chemistry have also made available novel compounds for testing in biochemical, physiological, and behavioral models of dystonia. Here, we briefly review motor circuit anatomy and the anatomical and functional abnormalities in dystonia. We then discuss recently identified therapeutic targets in dystonia based on recent preclinical animal studies and clinical trials investigating novel therapeutics.

Original languageEnglish
Article number104526
JournalNeurobiology of Disease
Volume130
DOIs
Publication statusPublished - Oct 1 2019

Fingerprint

Dystonia
Movement Disorders
Therapeutics
Essential Tremor
Body Regions
Pharmaceutical Chemistry
Dyskinesias
Muscle Contraction
Posture
Smooth Muscle
Parkinson Disease
Anatomy
Clinical Trials

Keywords

  • Anatomy
  • Animal models
  • Basal ganglia
  • Cerebellum
  • Drug discovery
  • Therapy

ASJC Scopus subject areas

  • Neurology

Cite this

The neurobiological basis for novel experimental therapeutics in dystonia. / Downs, Anthony M.; Roman, Kaitlyn M.; Campbell, Simone A.; Pisani, Antonio; Hess, Ellen J.; Bonsi, P.

In: Neurobiology of Disease, Vol. 130, 104526, 01.10.2019.

Research output: Contribution to journalReview article

Downs, Anthony M. ; Roman, Kaitlyn M. ; Campbell, Simone A. ; Pisani, Antonio ; Hess, Ellen J. ; Bonsi, P. / The neurobiological basis for novel experimental therapeutics in dystonia. In: Neurobiology of Disease. 2019 ; Vol. 130.
@article{74ecd01b70c3463f97a6d8bebb84bd01,
title = "The neurobiological basis for novel experimental therapeutics in dystonia",
abstract = "Dystonia is a movement disorder characterized by involuntary muscle contractions, twisting movements, and abnormal postures that may affect one or multiple body regions. Dystonia is the third most common movement disorder after Parkinson's disease and essential tremor. Despite its relative frequency, small molecule therapeutics for dystonia are limited. Development of new therapeutics is further hampered by the heterogeneity of both clinical symptoms and etiologies in dystonia. Recent advances in both animal and cell-based models have helped clarify divergent etiologies in dystonia and have facilitated the identification of new therapeutic targets. Advances in medicinal chemistry have also made available novel compounds for testing in biochemical, physiological, and behavioral models of dystonia. Here, we briefly review motor circuit anatomy and the anatomical and functional abnormalities in dystonia. We then discuss recently identified therapeutic targets in dystonia based on recent preclinical animal studies and clinical trials investigating novel therapeutics.",
keywords = "Anatomy, Animal models, Basal ganglia, Cerebellum, Drug discovery, Therapy",
author = "Downs, {Anthony M.} and Roman, {Kaitlyn M.} and Campbell, {Simone A.} and Antonio Pisani and Hess, {Ellen J.} and P. Bonsi",
year = "2019",
month = "10",
day = "1",
doi = "10.1016/j.nbd.2019.104526",
language = "English",
volume = "130",
journal = "Neurobiology of Disease",
issn = "0969-9961",
publisher = "Academic Press Inc.",

}

TY - JOUR

T1 - The neurobiological basis for novel experimental therapeutics in dystonia

AU - Downs, Anthony M.

AU - Roman, Kaitlyn M.

AU - Campbell, Simone A.

AU - Pisani, Antonio

AU - Hess, Ellen J.

AU - Bonsi, P.

PY - 2019/10/1

Y1 - 2019/10/1

N2 - Dystonia is a movement disorder characterized by involuntary muscle contractions, twisting movements, and abnormal postures that may affect one or multiple body regions. Dystonia is the third most common movement disorder after Parkinson's disease and essential tremor. Despite its relative frequency, small molecule therapeutics for dystonia are limited. Development of new therapeutics is further hampered by the heterogeneity of both clinical symptoms and etiologies in dystonia. Recent advances in both animal and cell-based models have helped clarify divergent etiologies in dystonia and have facilitated the identification of new therapeutic targets. Advances in medicinal chemistry have also made available novel compounds for testing in biochemical, physiological, and behavioral models of dystonia. Here, we briefly review motor circuit anatomy and the anatomical and functional abnormalities in dystonia. We then discuss recently identified therapeutic targets in dystonia based on recent preclinical animal studies and clinical trials investigating novel therapeutics.

AB - Dystonia is a movement disorder characterized by involuntary muscle contractions, twisting movements, and abnormal postures that may affect one or multiple body regions. Dystonia is the third most common movement disorder after Parkinson's disease and essential tremor. Despite its relative frequency, small molecule therapeutics for dystonia are limited. Development of new therapeutics is further hampered by the heterogeneity of both clinical symptoms and etiologies in dystonia. Recent advances in both animal and cell-based models have helped clarify divergent etiologies in dystonia and have facilitated the identification of new therapeutic targets. Advances in medicinal chemistry have also made available novel compounds for testing in biochemical, physiological, and behavioral models of dystonia. Here, we briefly review motor circuit anatomy and the anatomical and functional abnormalities in dystonia. We then discuss recently identified therapeutic targets in dystonia based on recent preclinical animal studies and clinical trials investigating novel therapeutics.

KW - Anatomy

KW - Animal models

KW - Basal ganglia

KW - Cerebellum

KW - Drug discovery

KW - Therapy

UR - http://www.scopus.com/inward/record.url?scp=85068760177&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85068760177&partnerID=8YFLogxK

U2 - 10.1016/j.nbd.2019.104526

DO - 10.1016/j.nbd.2019.104526

M3 - Review article

AN - SCOPUS:85068760177

VL - 130

JO - Neurobiology of Disease

JF - Neurobiology of Disease

SN - 0969-9961

M1 - 104526

ER -