Advances in CRISPR Gene Editing Offer Hope for Neurodevelopmental Disorders
TL;DR Summary
The article discusses the use of CRISPR activation techniques to address SCN2A-related neurodevelopmental disorders, with data available from RNA sequencing studies and various mouse models, aiming to develop targeted gene therapies.
- CRISPR activation for SCN2A -related neurodevelopmental disorders Nature
- CRISPR approach offers hope for severe childhood brain disorder Medical Xpress
- UCSF Researchers Use CRISPR to Restore SCN2A Gene Function in Preclinical Models geneonline.com
- Landmark UCSF Study Demonstrates Functional Rescue of SCN2A Loss of Function, Underscoring the Potential of Regel Therapeutics' Targeted EpiEditing Platform. WV News
- Harvard gene editing treatment corrects brain mutations and improves survival The Brighter Side of News
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