Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47
遗传性痉挛性截瘫47型AP4B1基因替代疗法的临床前开发
期刊:EMBO Molecular Medicine
影响因子:9
doi:10.1038/s44321-024-00148-5
Jessica P Wiseman, Joseph M Scarrott, João Alves-Cruzeiro, Afshin Saffari, Cedric Böger, Evangelia Karyka, Emily Dawes, Alexandra K Davies, Paolo M Marchi, Emily Graves, Fiona Fernandes, Zih-Liang Yang, Ian Coldicott, Jennifer Hirst, Christopher P Webster, J Robin Highley, Neil Hackett, Adrienn Angy