Gene therapy improves motor and mental function of aromatic l-amino acid decarboxylase deficiency

In patients with aromatic l-amino acid decarboxylase (AADC) deficiency, a decrease in catecholamines and serotonin levels in the brain leads to developmental delay and movement disorders. The beneficial effects of gene therapy in patients from 1 to 8 years of age with homogeneous severity of disease...

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Detalles Bibliográficos
Autores principales: Kojima, Karin, Nakajima, Takeshi, Taga, Naoyuki, Miyauchi, Akihiko, Kato, Mitsuhiro, Matsumoto, Ayumi, Ikeda, Takahiro, Nakamura, Kazuyuki, Kubota, Tetsuo, Mizukami, Hiroaki, Ono, Sayaka, Onuki, Yoshiyuki, Sato, Toshihiko, Osaka, Hitoshi, Muramatsu, Shin-ichi, Yamagata, Takanori
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2019
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6377184/
https://www.ncbi.nlm.nih.gov/pubmed/30689738
http://dx.doi.org/10.1093/brain/awy331

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6377184/
https://www.ncbi.nlm.nih.gov/pubmed/30689738
http://dx.doi.org/10.1093/brain/awy331

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