Modulation of the GDF11-SMAD3 signaling pathway to treat Rett syndrome
Modulating MeCP2 expression heterogeneity in brain cells for increasing gene therapy efficacy and safety in Rett syndrome
RNA Editing-Mediated Modification of a Common Nonsense Mutation in Rett Syndrome
Novel Neuromodulation Therapy to Address Sensory Hypersensitivity in Rett Syndrome
Targeting choroid plexus Otx2 as novel therapy in RTT
The interplay of the heat shock cascade and Rett syndrome
Leveraging the RNA binding activity of MECP2 to improve gene replacement therapy for Rett syndrome
A CRISPR toolbox for curative strategies for Rett syndrome
Anticodon-engineered tRNAs for the treatment of MECP2 nonsense mutations
Preclinical development of Rett syndrome ASO drugs
Development of treatment responsive biomarker in RTT
Evaluation of AAV9-BDNF treatment in two mouse models of RTT
