Methods and compositions for reconstituting microglia
AssigneesChildren's Medical Center Corporation, Dana-Farber Cancer Institute, Inc.
InventorsAlessandra Biffi, Annita Montepeloso
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Abstract
The present disclosure features CX3CR1 hemizygous and/or homozygous defective cells and methods of using such cells for the treatment of a metabolic or neurological disorder. The disclosed methods include methods for making and modifying CX3CR1 hemizygous and/or homozygous defective cells, such as hematopoietic stem progenitor cells. Other disclosed methods include methods of treating a subject having or suspected of having a metabolic or neurological disease comprising administering to the subject a composition comprising a hemizygous and/or homozygous defective CX3CR1 cell. The CX3CR1 hemizygous and/or homozygous defective cell may be modified to have a nucleic acid molecule encoding a therapeutic polypeptide or polynucleotide.
Claims14
A CX3CR1 haploinsufficent cell comprising an exogenous nucleic acid molecule inserted into one allele of a CX3CR1 gene, wherein the insertion disables the one allele of the CX3CR1 gene.
Dependencies are inferred from the claim text.