公开(公告)号：EP1814984A2

公开(公告)日：2007-08-08

申请号：EP05816095.3

申请日：2005-09-19

申请人： Biomarin Pharmaceutical Inc. ,  THE SCRIPPS RESEARCH INSTITUTE

发明人： GAMEZ, Alejandra ,  WANG, Lin ,  KIM, Woomi ,  STRAUB, Mary ,  PATCH, Marianne, G. ,  KAKKIS, Emil ,  OPPENHEIMER, Dan ,  FITZPATRICK, Paul, A. ,  HEFT, Robert ,  STEVENS, Raymond, C.

IPC分类号： C12N9/88

CPC分类号： C12N9/88 ,  C07K2299/00 ,  Y02A90/26

摘要： The present invention pertains to the use of the protein phenylalanine ammonia-lyase, as well as the biologically-active derivatives of the said protein for preventing or treating diseases associated with a phenylalanine imbalance in a human or animal body. More particularly, the present invention relates to the therapeutic use of the above-cited molecules for preventing or treating a phenylalanine imbalance in vivo. This invention also deals with therapeutic compositions comprising a pharmaceutically active amount of the above-described therapeutic molecules as well as with therapeutic methods using the said therapeutic compositions. Finally, the present invention relates to processes for selecting more therapeutically-effective variants of said protein as well as to the selected variants themselves.