Distillery Therapeutics


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<h1 class="text title distillery_title" doc-editable="text" data-component-id="doc-1ef7l992d0">CRISPR-based FKRP correction for a broad spectrum of muscular dystrophies</h1><div class="byline ng-tns-c17-4 ng-star-inserted" data-component-id="doc-1ef7l9he70">
    <span class="ng-tns-c17-4" doc-editable="text">By biocentury staff</span>
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MDlMU25OU0VrUjVQVlFtaWZjTDlWQlBmS3R5Uzh0QkFSSlp2RTBPK1lNQ1ZibXlhb1lOeWd1RnY5TjFnZVN2T0tadHpzMVVTazVOZ0s4RWZVL1BFM1RvZ0RPdm9MSjFmRGFydnJUdndRWHRacTRlTHFkUHduUXJWZVVpaHFGNCsrOFY3SnE4a0JmWEU1cGpOQ2REbXFmZDRIdVZSWG1WYlcxVUpHMnlqOWpPNmNqQlE9PQ==

DISEASE CATEGORY: MusculoskeletalINDICATION: Muscular dystrophy......

Musculoskeletal

Muscular dystrophy

Fukutin related protein (FKRP)

CRISPR-associated protein 9 (Cas9)

CRISPR-based FKRP correction for a broad spectrum of muscular dystrophies

DISEASE CATEGORY: Musculoskeletal<br/>INDICATION: Muscular dystrophy Replacing the mutant fukutin-related protein (FKRP) gene with the wild-type sequence using CRISPR-Cas9 technology could treat

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