Columbia University in the City of New York · PhysiologyThe MECP2 Initiative
X. Shawn Liu · New York, New York
Research Focus
Dr. X. Shawn Liu's lab at Columbia University focuses on developing innovative therapies for Rett syndrome, a severe neurodevelopmental disorder primarily affecting girls. The lab's research aims to reactivate the MECP2 gene from a silenced X chromosome, which offers a potential treatment by restoring function in neurons. They utilize advanced mouse models and genetic editing tools to explore this groundbreaking approach, with hopes of leading to future clinical trials.
Rett syndromeMECP2gene therapyepigenetic editinganimal models
Research Aims
5AIM 01Develop therapies for Rett syndrome by targeting MECP2 reactivation.
AIM 02Utilize advanced transgenic models to assess treatment effectiveness.
AIM 03Investigate the impact of MECP2 reactivation on neuronal function.
AIM 04Explore innovative delivery methods for gene editing tools.
AIM 05Conduct preclinical studies that could inform future clinical trials.
Skills the Lab Values
CRISPRtransgenic mouse modelsDNA methylation editinggene expression analysisin vivo testing
Output against the department
Publications67bottom 13% of dept.248th of 286
this lab
most publishedleast published
Citations6.6ktop 49% of dept.141st of 286
this lab
most citedleast cited
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