Treatment of canavan disease
AssigneeCity of Hope
InventorsYanhong SHI, Lizhao FENG, Jianfei CHAO, Guihua SUN
Life of this patent
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Abstract
Disclosed herein are methods of treating Canavan disease in a subject through restoring ASPA enzymatic activities in the subject by expressing an exogenous functional ASPA gene in the brain of the subject. Also disclosed are a process of producing neural precursor cells, including NPCs, glial progenitor cells and OPCs, which express an exogenous functional ASPA gene and the neural precursor cells produced by this process.
Claims27
1-32. (canceled)
A method for treating Canavan disease in a subject, comprising:
- reprogramming or converting somatic cells isolated from the subject into induced pluripotent stem cells (iPSCs);
- differentiating the iPSCs into neural precursor cells;
- introducing a functional ASPA gene having a R132G mutation (R132G ASPA) into the neural precursor cells to obtain genetically corrected neural precursor cells which express the R132G ASPA;
- and
- transplanting the genetically corrected neural precursor cells into the brain of the subject.
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A method of producing ASPA neural precursor cells, comprising:
- reprogramming or converting somatic cells isolated from a subject suffering from Canavan disease into induced pluripotent stem cells (iPSCs);
- differentiating the iPSCs into neural precursor cells;
- and
- introducing a functional ASPA gene having a R132G mutation (R132G ASPA) in the neural precursor cells to obtain genetically corrected neural precursor cells which express R132G ASPA.
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Neural precursor cells which express an exogenous functional ASPA gene produced by a process comprising the steps of:
- reprogramming or converting somatic cells isolated from a subject suffering from Canavan disease into induced pluripotent stem cells (iPSCs),
- differentiating the iPSCs into neural precursor cells;
- and
- introducing a functional ASPA gene having a R132G mutation (R132G ASPA) in the neural precursor cells to obtain genetically corrected neural precursor cells which express R132G ASPA.
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Dependencies are inferred from the claim text.