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Updated: Jun 9, 2026
10:47
Rapid Detection of Neurodevelopmental Phenotypes in Human Neural Precursor Cells (NPCs)
Published on: March 2, 2018
Emerging pharmacotherapies for neurodevelopmental disorders
Daniel Z Wetmore1, Craig C Garner
1Department of Psychiatry and Behavioral Sciences, Nancy Pritzker Laboratory, Stanford University, Stanford, CA 94305-5485, USA.
Journal of Developmental and Behavioral Pediatrics : JDBP
|September 4, 2010
Summary
Translational neuroscience research in genetic neurodevelopmental disorders (NDDs) uses animal models to uncover dysfunction mechanisms. Promising pharmacotherapies targeting common pathways show potential for treating cognitive deficits in humans.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Neurodevelopmental disorders (NDDs) with genetic origins are a significant research focus.
- Transgenic animal models are crucial for understanding disease mechanisms and testing interventions.
Purpose of the Study:
- To review translational research on genetic NDDs using animal models.
- To highlight pharmacotherapeutic strategies for conditions like Down syndrome, fragile X, Rett syndrome, NF1, TSC, and autism.
Main Methods:
- Utilizing transgenic animal models that replicate human genetic insults.
- Analyzing common pathways and mechanisms of dysfunction across various NDDs.
- Evaluating pharmacotherapeutic strategies for brain function deficits.
Main Results:
- Animal models reveal that cognitive deficits in NDDs can be addressed even in adult subjects.
- Common mechanisms include abnormal gene dosage, neurotransmitter imbalances, and neuronal circuit deficits.
- Multiple brain systems and behaviors affected by NDDs may respond to targeted drug therapies.
Conclusions:
- Translational research aims to develop causal pharmacotherapies for NDDs, moving beyond symptomatic treatments.
- Several emerging therapeutic strategies show promise for clinical application in humans.
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