Transplanting stem cell-derived microglia to treat leukodystrophies

A method of treating or preventing a leukodystrophy in a subject having a gene encoding a mutant Colony Stimulating Factor-1 Receptor (CSF1R) protein, the method comprising:

• (a) obtaining a cell from a donor;
• (b) producing a cell comprising a functional CSF1R protein from the cell obtained from the donor, wherein the producing comprises:
• (i) repairing or replacing at least a portion of a gene encoding a mutant CSF1R protein in the cell obtained from the donor;
• or
• (ii) culturing the cell obtained from the donor in a microglial differentiation medium for no more than 14 days;
• and
• (c) transplanting the cell comprising the functional CSF1R protein into the subject, thereby treating or preventing the leukodystrophy in the subject.

A method of preparing a cell therapy for treating or preventing leukodystrophy, the method comprising:

• (n) obtaining a cell from a donor, wherein the donor is the subject or a healthy individual;
• and
• (o) producing a cell comprising a functional CSF1R protein from the cell obtained from the doner, wherein producing comprises (i) repairing or replacing a gene encoding a mutant CSF1R protein in the cell obtained from the donor, or (ii) culturing the cell obtained from the donor in a differentiation medium for no more than 14 days,
• thereby preparing the cell therapy.