专利检索 ap:("BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM") AND inv:"Sergey D. EGRANOV" 第 1 页

1.

发明公开
MODIFIED NUCLEIC ACID COMPOSITIONS AND ASSOCIATED METHODS FOR TREATMENT OF PHENYLKETONURIA 审中-公开

公开(公告)号：US20240342207A1

公开(公告)日：2024-10-17

申请号：US18294680

申请日：2022-08-02

申请人： BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM

发明人： Liuqing YANG , Chunru LIN , Sergey D. EGRANOV , Yajuan LI

IPC分类号： A61K31/7105 , A61K48/00 , A61P3/00 , C12N15/11

CPC分类号： A61K31/7105 , A61K48/0075 , A61P3/00 , C12N15/11 , C12N2310/315 , C12N2310/322 , C12N2310/3517

摘要： This disclosure provides modified nucleic acids and compositions thereof that are able to mimic the human HULC lncRNA and/or the mouse Pair lncRNA and enhance phenylalanine hydroxylase enzyme (PAH)-substrate binding affinity, PAH-cofactor binding affinity, and/or PAH enzymatic conversion of phenylalanine to tyrosine. Also provided are methods of using the modified nucleic acids and compositions thereof to treat subjects having or suspected of having phenylketonuria and hyperphenylalaninemia.