Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA
抗淀粉样蛋白治疗对神经病性粘多糖病具有广泛疗效,并与 MPS-IIIA 的基因治疗产生协同作用
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2024.09.030
Marianna Giaccio, Antonio Monaco, Laura Galiano, Andrea Parente, Luigi Borzacchiello, Riccardo Rubino, Frank-Gerrit Klärner, Dennis Killa, Claudia Perna, Pasquale Piccolo, Marcello Marotta, Xuefang Pan, Marie Khijniak, Ibrar Siddique, Thomas Schrader, Alexey V Pshezhetsky, Nicolina Cristina Sorrenti