Clive N. Svendsen

TL;DR: Results indicate that motor neurons in presymptomatic G93A animals are not affected by an invasive puncture wound injury involving reactive astrocytes, and acute trauma alone does not accelerate disease onset or progression in this ALS model, important for future strategies of gene and cell therapies directly targeting the spinal cord of ALS patients.

TL;DR: Intravenous administration of AAV9, carrying the human MCT8, to juvenile dKO mice manifesting Allan-Herndon-Dudley syndrome has long-term a beneficial effect predominantly on the central nervous system.

TL;DR: This 3-cell neurogenic scheme echoes observations in rodents in vivo and in human fetal slice cultures in vitro, providing evidence that hNPCs represent a renewable and robust in vitro assay system to explore mechanisms of human neurogenesis without the continual need for fresh primary human fetal tissue.

TL;DR: The production of a mifepristone-inducible vector system for regulated expression of transgenes within the CNS is described for use in the development of safe and efficient gene therapy for neurological disorders.

TL;DR: This xenograft study with cyclosporine-treated animals demonstrates that readministration of neural progenitors into the fellow eye did not induce anti-graft immune responses or lower therapeutic efficacy of hNPCctx in preserving vision, suggesting readministrations of progenitor cells to sustain long-term efficacy may be an option for long- term therapies of retinal degeneration.