Detalhe da pesquisa
1.
Intragenic complementation of amino and carboxy terminal SMN missense mutations can rescue Smn null mice.
Hum Mol Genet
; 29(21): 3493-3503, 2020 11 01.
Artigo
Inglês
| MEDLINE | ID: mdl-33084884
2.
Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice.
Neurobiol Dis
; 159: 105488, 2021 11.
Artigo
Inglês
| MEDLINE | ID: mdl-34425216
3.
Base editing rescue of spinal muscular atrophy in cells and in mice.
Science
; 380(6642): eadg6518, 2023 04 21.
Artigo
Inglês
| MEDLINE | ID: mdl-36996170