Compositions and methods for treating diseases and disorders of the central nervous system
AssigneesChildren's Medical Center Corporation, Ospedale San Raffaele S.R.L., Politecnico Di Milano, Fondazione Telethon Ets, Dana-Farber Cancer Institute, Inc.
InventorsAlessandra Biffi, Marco Peviani, Davide Moscatelli, Alessia Capotondo, Rita Milazzo, Umberto Capasso Palmiero
Life of this patent
Estimating the term…
Abstract
The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.
Claims8
A method of delivering a Hematopoietic Stem Progenitor Cell (HSPC) to a human subject that has or is at increased risk of developing a lysosomal storage disorder or neurodegenerative disease, the method comprising administering to the subject a human CD34+ Hematopoietic Stem Progenitor Cell by Intra-cerebral Ventricular Injection (ICV), wherein the subject was previously treated with a cytotoxic agent or radiation in connection with the ablation of microglia and/or their progenitors wherein the cytotoxic agent is an alkylating agent, and the administration reconstitutes microglia by HSPC engraftment in the subject.
A method of delivering a Hematopoietic Stem Progenitor Cell (HSPC) to a human subject that has or is at increased risk of developing a lysosomal storage disorder or neurodegenerative disease, the method comprising administering to the subject a human CD150+ CD48−, Hematopoietic Stem Progenitor Cell by Intra-cerebral Ventricular Injection (ICV), wherein the subject was previously treated with a cytotoxic agent or radiation in connection with the ablation of microglia and/or their progenitors wherein the cytotoxic agent is an alkylating agent, and the administration reconstitutes microglia by HSPC engraftment in the subject.
A method of delivering a Hematopoietic Stem Progenitor Cell (HSPC) to a subject that has or is at increased risk of developing a lysosomal storage disorder or neurodegenerative disease, the method comprising administering to the subject a Lin-Hematopoietic Stem Progenitor Cell by Intra-cerebral Ventricular Injection (ICV), wherein the subject was previously treated with a cytotoxic agent or radiation in connection with the ablation of microglia and/or their progenitors wherein the cytotoxic agent is an alkylating agent, and the administration reconstitutes microglia by HSPC engraftment in the subject.
Dependencies are inferred from the claim text.