abstract |
The present invention provides a polynucleotide comprising a UBE3A open reading frame (ORF), a vector comprising the same, and a vector comprising the same, and using the same to deliver the ORF to a cell or individual, and aberrant expression of the UBE3A gene or the UBE3A gene in the subject, such as Angelman's syndrome It relates to a method of treating a disease associated with aberrant activity of the construct. |