Genetically modulated substrate reduction therapy

The concept of enzyme replacement as a potential therapeutic approach to ameliorate lysosomal storage disorders (LSDs) is virtually as old as the concept of LSD itself. In fact, right after the first enzymatic deficiency underlying a LSD was described, both concepts were established and, for years,...

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Detalhes bibliográficos
Autor principal: Coutinho, Maria Francisca (author)
Outros Autores: Santos, Juliana Inês (author), Matos, Liliana (author), Alves, Sandra (author)
Formato: conferenceObject
Idioma:eng
Publicado em: 2018
Assuntos:
Texto completo:http://hdl.handle.net/10400.18/5516
País:Portugal
Oai:oai:repositorio.insa.pt:10400.18/5516