Modified UBE3A gene for a gene therapy approach for Angelman syndrome

A ubiquitin protein ligase E3A (UBE3A) vector comprising:

• a transcription initiation sequence wherein the transcription initiation sequence is a cytomegalovirus chicken-beta actin hybrid promoter, or human ubiquitin c promoter,
• a UBE3A sequence disposed downstream of the transcription initiation sequence, wherein the UBE3A sequence is SEQ ID NO:
• 12, SEQ ID NO:
• 13, SEQ ID NO:
• 15, a cDNA encoding SEQ ID NO:
• 7, a cDNA encoding SEQ ID NO:
• 14, or a nucleotide sequence possessing at least 90% sequence identity thereto;
• a secretion sequence disposed downstream of the transcription initiation sequence and upstream of the UBE3A sequence, wherein the secretion sequence is SEQ ID NO:
• 2, SEQ ID NO:
• 8, SEQ ID NO:
• 9, SEQ ID NO:
• 10, a cDNA encoding SEQ ID NO:
• 3, or a homologous sequence;
• and
• a cell uptake sequence disposed downstream of the transcription initiation sequence and between the secretion sequence and the UBE3A sequence, wherein the cell uptake sequence is SEQ ID NO:
• 4, a cDNA encoding SEQ ID NO:
• 5, SEQ ID NO:
• 11, or a homologous sequence;
• wherein the vector is an adeno-associated viral (AAV) vector.