Neuroinflammation, characterized by microglial cell-activation, plays a major role in the pathogenesis and progression of multiple neurodegenerative diseases, such as Lysosomal Storage Disorders and Alzheimer’s, severe human conditions currently lacking an effective treatment.
Ex vivo gene therapy based on genetically modified hematopoietic stem cells (HSCs) approach shows great promise, yet the beneficial effect of it relies on CNS-associated microglia-like cells derived from the transplanted HSCs due to their role in neuroinflammation reduction after treatment.
To increase the efficacy of such therapeutic strategy, we have developed a proprietary synthetic promoter for a gene transfer vector that allows for a controlled –low basal levels that are significantly increased upon inflammation– expression of therapeutic transgenes in immune-like cells.
The inducible promoter sequence is validated both in vitro and in vivo.

TRL

Lead Optimization/Preclinical

Inventors

Alessandra
Biffi

Valentina
Poletti

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