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Method and material for a selective Engrafting in the Central Nervous System

Microglia AblationnanomedicineNanoparticlesSelective Drug DeliveryStem Cell TransplantationTargeted therapy


Compositions and methods for the treatment or prevention of neurological diseases or disorders of the CNS (Central Nervous System), for example neurodegenerative diseases, by reconstituting brain myeloid cells and microglia. Specifically, a method for efficiently and selectively engrafting CNS cells with characteristics of microglia, including microglia progenitor cells, for therapeutic purposes and for ablation in the brain of resident myeloid cells, with proliferation capacity. These include nanoparticles that target microglia and / or microglia progenitors.

Technical features

The invention also provides compositions and methods for ablation and reconstitution of microglia. This methodology could be employed to achieve successful, timely and, in the case of exclusive CNS involvement, selective CNS engraftment of the transplanted cells in the brain and acquisition of myeloid/microglia features for delivery of therapeutic molecules and/or modulation of myeloid/microglia features upon partial renewal of the cell pool. Compared to existing technologies, there is no need of lyophilization and dialysis to store the formulated lipophilic drug, since the new polymeric surfactant is able to self-assembly directly into NPs (nanoparticles) just through the use of a syringe and a filter syringe. The formulation can be reconstituted directly at the bed of the patient before injection. In addition, the polymeric surgactant is completely biocompatible and biodegradable. In addition, these NPs can deliver these cytoxic agents to microglial cells via Intra-cerebral Ventricular Injection (ICV). In this way, it is possible to ablate the microglial cells and/or progenitors for the treatment of lysosomal storage disorders.

Possible Applications

  • NPs-mediated selective targeting of reactive microglia cells could represent a promising therapeutic approach for several neurodegenerative diseases, including Alzheimer’s disease or Amyotrophic Lateral Sclerosis
  • Treatment or prevention of neurological diseases (e.g., lysosomal storage disorder) by reconstitution of microglia upon transplantation of hematopoietic stem cells


  • Cell-selective drug release in the central nervous system (CNS)
  • Lower toxicity of the drug loaded nanoparticles compared to the free clinical grade drug.
  • Brain-specific conditioning regimens that allow for a selective brain engraftment upon hematopoietic stem cell transplantation.