Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
临床前慢病毒造血干细胞基因疗法可纠正庞贝病相关的肌肉和神经系统症状
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2024.09.024
Yoon John K, Schindler Jeffrey W, Loperfido Mariana, Baricordi Cristina, DeAndrade Mark P, Jacobs Mary E, Treleaven Christopher, Plasschaert Robert N, Yan Aimin, Barese Cecilia N, Dogan Yildirim, Chen Vicky Ping, Fiorini Claudia, Hull Fritz, Barbarossa Luigi, Unnisa Zeenath, Ivanov Daniel, Kutner Robert H, Guda Swaroopa, Oborski Christine, Maiwald Tim, Michaud Véronique, Rothe Michael, Schambach Axel, Pfeifer Richard, Mason Chris, Biasco Luca, van Til Niek P
