GENE THERAPY EXPRESSION SYSTEM ALLEVIATING CARDIAC TOXICITY OF FKRP

    公开(公告)号:US20220370640A1

    公开(公告)日:2022-11-24

    申请号:US17753768

    申请日:2020-09-18

    Abstract: The present invention concerns an expression system for systemic administration comprising a sequence encoding a FKRP protein, and: —a promoter sequence allowing the expression at a therapeutically acceptable level of FKRP in the skeletal muscles and a target sequence of an miRNA expressed in the heart; or—a promoter sequence allowing the expression at a therapeutically acceptable level of FKRP in the skeletal muscles and presenting a promoter activity at a toxically acceptable level in the heart; and its use for the treatment of various diseases linked to FKRP deficiencies.

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