[EN] SOLUBLE COMPOUNDS FOR IMPROVED GENE THERAPY METHODS<br/>[FR] COMPOSÉS SOLUBLES POUR MÉTHODES AMÉLIORÉES DE THÉRAPIE GÉNIQUE
申请人:BLUEBIRD BIO INC
公开号:WO2014015318A1
公开(公告)日:2014-01-23
The present disclosure provides methods and compositions for improving the efficiency of cell therapy and gene delivery to cells employing compounds that modulate the peroxisome proliferator activated receptor (PP AR) signaling pathway. More particularly, methods and materials for useful for safely and reliably improving the efficiency of methods for homing or engraftment of cells, such as human hernatopoietic.stem cells (HSC), and for improving the efficiency of transducing such cells with viruses and/or viral vectors are provided. The compositions and methods are useful for therapeutic indications amenable to treatment with cell therapies and gene therapies.