Abstract
Genetic neurodevelopmental disorders are characterized by abnormal neurophysiological and behavioral phenotypes, affecting individuals worldwide. While the subject has been heavily researched, current treatment options relate mostly to alleviating symptoms, rather than targeting the altered genome itself. In this review, we address the neurogenetic basis of neurodevelopmental disorders, genetic tools that are enabling precision research of these disorders in animal models, and postnatal gene-therapy approaches for neurodevelopmental disorders derived from preclinical studies in the laboratory.
| Original language | English |
|---|---|
| Pages (from-to) | 414-422 |
| Number of pages | 9 |
| Journal | Neural Regeneration Research |
| Volume | 16 |
| Issue number | 3 |
| DOIs | |
| State | Published - Mar 2021 |
UN SDGs
This output contributes to the following UN Sustainable Development Goals (SDGs)
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SDG 3 Good Health and Well-being
Keywords
- CRISPR-Cas9
- adeno-associated virus
- developmental window
- genetic therapy
- mouse models
- neurodevelopmental disorders
- non-human primates
- preclinical studies
All Science Journal Classification (ASJC) codes
- Developmental Neuroscience
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