Terapia gênica individualizada: a experiência da paraparesia espástica hereditária tipo 50

Referências

1. Dowling JJ, Pirovolakis T, Devakandan K, et al. AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient. Nat Med. June 2024. doi:10.1038/s41591-024-03078-4 

2. de Souza CFM, Alves T, Duarte J, et al. Interim results from the first-in-human intracisternal dosing of RGX-181 investigational AAV9 gene therapy in a child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Molecular Genetics and Metabolism. 2024;141(2):107970. doi:10.1016/j.ymgme.20