Year: 2021
-
Towards targeted neutralization of the disease causing SVA in XDP brain organoids
-
Novel AAV-compatible CRISPR gene editors for XDP SVA excision gene therapy
-
Investigating RNA- and RNA-binding protein mediated toxicity in iPSC-derived XDP cell culture models to potentially identify shared mechanisms with other dystonia types and neurodegenerative diseases
-
Profiling chromatin regulation of the XDP-specific SVA insertion by targeted proximity labelling and epigenomics
-
Novel mouse models for the analysis of XDP
-
Promise and challenges of dystonia brain banking: establishing a human tissue repository for studies of X-Linked Dystonia-Parkinsonism
-
Speech and swallowing deficits in X-Linked Dystonia-Parkinsonism
-
Striatal Cholinergic Dysregulation after Neonatal Decrease in X-Linked Dystonia-Parkinsonism-Related TAF1 Isoforms
-
Unbiased identification of novel transcription factors in striatal compartmentation and striosome maturation