Title:
COMPOSITIONS AND METHODS FOR TREATING PHENYLKETONURIA
Document Type and Number:
Japanese Patent JP2023068051
Kind Code:
A
Abstract:
To develop an alternative method for treatment of phenylketonuria.SOLUTION: In an aspect, a viral vector is disclosed. The viral vector comprises a phenylalanine hydroxylase (PAH) sequence for expressing at least one of PAH and variants thereof, where the PAH sequence is truncated. In embodiments, the PAH sequence is truncated at a 3' untranslated region (UTR) of the sequence. In embodiments, the PAH sequence comprises at least one of 80%, 85%, 90%, 95% and 100% identity to at least one of SEQ ID NO: 2, SEQ ID NO: 3 and SEQ ID NO: 4.SELECTED DRAWING: Figure 12
Inventors:
Taylor Rafusen
Charles David Pausa
Charles David Pausa
Application Number:
JP2023043194A
Publication Date:
May 16, 2023
Filing Date:
March 17, 2023
Export Citation:
Assignee:
American Gene Technologies International Incorporated
International Classes:
C12N15/867; A61K35/76; A61K38/02; A61K48/00; A61P13/02; C12N7/01; C12N15/113; C12N15/53
Attorney, Agent or Firm:
Shusaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto