Chenodeoxycholic acid rescues axonal degeneration in induced pluripotent stem cell-derived neurons from spastic paraplegia type 5 and cerebrotendinous xanthomatosis patients.
Yongchao MouGhata A NandiSukhada MukteEric ChaiZhenyu ChenJorgen E NielsenTroels T NielsenChiara CriscuoloCraig BlackstoneMatthew J FraidakisXue-Jun LiPublished in: Orphanet journal of rare diseases (2023)
Our data demonstrate disease-specific lipid disturbances and axonopathy mechanisms in human pluripotent stem cell-based neuronal models of CTX and SPG5 and identify CDCA, an established treatment of CTX, as a potential pharmacotherapy for SPG5. We propose this novel treatment strategy to rescue axonal degeneration in SPG5, a currently incurable condition.
Keyphrases
- stem cells
- end stage renal disease
- spinal cord injury
- chronic kidney disease
- endothelial cells
- spinal cord
- prognostic factors
- newly diagnosed
- klebsiella pneumoniae
- ejection fraction
- drug induced
- smoking cessation
- electronic health record
- escherichia coli
- blood brain barrier
- risk assessment
- brain injury
- mesenchymal stem cells
- diabetic rats
- deep learning
- replacement therapy
- patient reported
- subarachnoid hemorrhage
- high glucose