THE UNIVERSITY COURT OF THE UNIVERSITY OF GLASGOW;THE UNIVERSITY COURT OF THE UNIVERSITY OF EDINBURGH
发明人:
BIRD, ADRIAN,TILLOTSON, REBEKAH,COBB, STUART ROBERT,HECTOR, RALPH DAVID
申请号：
CA3057425
公开号：
CA3057425A1
申请日:
2018.03.23
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
MeCP2 based therapy. The present invention relates to synthetic polypeptides that are useful in the treatment of disorders associated with reduced MeCP2 activity, including Rett syndrome. The present invention provides synthetic polypeptides comprising: i) an MBD amino acid sequence showing at least 70% similarity with the amino acid sequence as depicted in SEQ ID NO: 1； and ii) an NID amino acid sequence showing at least 70% similarity with the amino acid sequence as depicted in SEQ ID NO: 2, wherein the polypeptide has a deletion of at least 50 amino acids, when compared to the full length MeCP2 e1 and e2 sequences. The invention further provides nucleic acid constructs, expression vectors, virions, pharmaceutical compositions, and cells providing polynucleotides of the invention. The invention further provides methods of treating or preventing disease in an animal comprising administering to said animal a synthetic polypeptide according to the invention.
