CORDIS Project
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This project focuses on developing a novel gene therapy for inherited neurometabolic diseases by targeting hematopoietic stem cells. It aims to correct genetic defects at a specific microglia locus to enhance the timely production of therapeutic microglia-like cells, potentially improving treatment outcomes for patient…
Hematopoietic stem cell (HSC) gene therapy based on self-inactivating integrating vectors has proven unprecedented therapeutic potential in inherited neurometabolic diseases (NMDs).
However, phenotypic effects are delayed after treatment likely due to the slow replacement of resident microglia by transplant-derived cells that hampers the broad application of this approach.
Moreover, unregulated gene expression driven by the in-use promoters could in the long term cause unwanted effects.
Finally,…
UNIVERSITA DEGLI STUDI DI PADOVA
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