Compositions and Methods for Treatment of Mitochondrial Diseases and for Differentiation of Cells to Neurons

摘要

Methods and compositions are provided for the treatment of a mitochondrial disease in an individual with the mitochondrial disease. Aspects of the methods include administering an inhibitor of a Pumilio-like protein and/or an inhibitor of a serine/arginine-rich family of pre-mRNA splicing factor (SR) protein to a subject. Also provided are methods, compositions, systems and kits for transdifferentiating target cells to neurons, which find use in producing neurons for the development of new therapies, for experimental evaluation, as a source of lineage- and cell-specific products, and the like, for example, for use in treating human disorders of the CNS.

主权项

1. A method of treating mitochondrial disease in a subject having a mitochondrial disease, comprising:
administering a therapeutically effective amount of an inhibitor of a Pumilio-like protein and/or an inhibitor of an SR protein to the subject.