Title:
POLYNUCLEOTIDES FOR THE TREATMENT OF DISEASE ASSOCIATED WITH GCASE DEFICIENCY
Document Type and Number:
WIPO Patent Application WO/2024/074142
Kind Code:
A1
Abstract:
Provided are codon-optimized GBA1polynucleotides that encode a GCase protein, with a portion of the coding sequence deviating from the wild-type. Additionally provided are expression constructs, vectors, viral particles, or compositions containing the disclosed polynucleotide. Furthermore, provided are methods and applications of these polynucleotides, expression constructs, vectors, viral particles, or compositions, including treatment of the disease or condition which is associated with GCase deficiency.
Inventors:
LIN QING (CN)
CHEN YIXIONG (CN)
SHENG XIAOJING (CN)
JIANG TIANQI (CN)
WU QIANG (CN)
CHEN YIXIONG (CN)
SHENG XIAOJING (CN)
JIANG TIANQI (CN)
WU QIANG (CN)
Application Number:
PCT/CN2023/123281
Publication Date:
April 11, 2024
Filing Date:
October 07, 2023
Export Citation:
Assignee:
LINGYI BIOTECH CO LTD (CN)
International Classes:
C12N5/0783; A61K38/47; C07K14/725
Attorney, Agent or Firm:
LIFANG & PARTNERS (CN)
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