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 language | English |
|---|---|
| Article number | 104526 |
| Journal | Neurobiology of Disease |
| Volume | 130 |
| DOIs | |
| Publication status | Published - Oct 1 2019 |
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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 journal › Review article
}
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 -