abstract |
The present invention presents a polynucleotide containing an optimized FIG4 open reading frame (ORF) sequence, a vector containing it, and an abnormality of the FIG4 gene for delivery of the ORF to a cell or subject and in the subject. It relates to a disorder associated with expression or aberrant activity of the FIG4 gene product, such as its use for treating CMT4J. [Selection diagram] Fig. 1 |