The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Aug. 20, 2024
Filed:
Jan. 25, 2019
Tokushima University, Tokushima, JP;
Jichi Medical University, Tokyo, JP;
Gene Therapy Research Institution Co., Ltd., Kanagawa, JP;
Kohji Itoh, Tokushima, JP;
Daisuke Tsuji, Tokushima, JP;
Shinichi Muramatsu, Tochigi, JP;
Katsuhito Asai, Kanagawa, JP;
TOKUSHIMA UNIVERSITY, Tokushima, JP;
JICHI MEDICAL UNIVERSITY, Tokyo, JP;
GENE THERAPY RESEARCH INSTITUTION CO., LTD., Kanagawa, JP;
Abstract
According to a conventional method for treatment of Sandhoff disease and Tay-Sachs disease comprising administering a modified β-subunit to a patient in the form of a protein, it is necessary that administration be performed frequently. This invention relates to a recombinant adeno-associated virus virion comprising: capsomere comprising a protein capable of forming a virus virion; and a polynucleotide packaged in the capsomere comprising a promoter sequence and nucleotide sequences operably linked to the promoter sequence encoding a first amino acid sequence derived from the amino acid sequence of the β-subunit of wild-type human β-hexosaminidase composed of amino acids 55 to 556 in the sequence as shown in SEQ ID NO: 28 by substitution of amino acids 312 to 318 with glycine, serine, glutamic acid, proline, serine, glycine, and threonine in that order and a second amino acid sequence, which is an amino acid sequence of a signal peptide linked to the N terminus of the first amino acid.