Antonio Fontanellas Romá,Gloria González Aseguinolaza,Maria Sol Rodriguez Pena,Maria Astrid Pañeda Rodriguez,Jaap Twisk,Jesús Maria Prieto Valtueña,Harald Petry,Sander Jan Hendrik Van Deventer
申请号：
US13121532
公开号：
US08697665B2
申请日:
2009.09.29
申请国别(地区):
US
年份:
2014
代理人:
摘要：
The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.
