Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia
开发一种口服治疗方法,用于挽救家族性自主神经功能紊乱表型小鼠模型中的步态共济失调和视网膜变性
期刊:American Journal of Human Genetics
影响因子:8.1
doi:10.1016/j.ajhg.2023.01.019
Elisabetta Morini, Anil Chekuri, Emily M Logan, Jessica M Bolduc, Emily G Kirchner, Monica Salani, Aram J Krauson, Jana Narasimhan, Vijayalakshmi Gabbeta, Shivani Grover, Amal Dakka, Anna Mollin, Stephen P Jung, Xin Zhao, Nanjing Zhang, Sophie Zhang, Michael Arnold, Matthew G Woll, Nikolai A Naryshk