Methods and compositions for the treatment of neurologic disease
AssigneesSangamo Therapeutics, Inc., Regents of the University of Minnesota
InventorsRussell DeKelver, R. Scott McIvor, Li Ou, Thomas Wechsler, Chester B. Whitley, Kanut Laoharawee
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Abstract
Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic IDUA or IDS protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of MPS I or MPS II disease.
Claims15
A method of reducing or preventing central nervous system (CNS) impairment in a subject with mucopolysaccharidosis type I (MPS I) or mucopolysaccharidosis type I (MPS II), the method comprising
- administering to the subject a promoterless donor vector comprising a transgene encoding human α-L-iduronidase (hIDUA) or human iduronate-2-sulftase (hIDS);
- and
- administering an expression vector encoding a pair of zinc finger nucleases (ZFNs) targeted to an endogenous albumin gene,
- wherein the transgene is integrated into the albumin gene in a liver cell following cleavage of the albumin gene such that the encoded hIDUA or hIDS is expressed and secreted from the liver and further wherein the hIDUA or hIDS secreted from the liver is found in the central nervous system (CNS) of the subject such that the CNS impairment is reduced or prevented.
A Hep2G cell comprising a promoterless donor vector encoding hIUDA or hIDS integrated into intron 1 of an albumin gene, wherein the hIUDA or hIDS is expressed and secreted from the Hep2G cell.
Dependencies are inferred from the claim text.