Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy.

Nasir, Gibran; Chopra, Rajiv; Elwood, Fiona; Ahmed, Seemin S

Nasir, Gibran; Chopra, Rajiv; Elwood, Fiona; Ahmed, Seemin S.

Afiliação

Nasir G; Department of Neuroscience, Novartis Institutes for BioMedical Research (NIBR), Cambridge, MA, United States.
Chopra R; AllianThera Biopharma, Boston, MA, United States.
Elwood F; Department of Neuroscience, Novartis Institutes for BioMedical Research (NIBR), Cambridge, MA, United States.
Ahmed SS; Department of Neuroscience, Novartis Institutes for BioMedical Research (NIBR), Cambridge, MA, United States.

Front Med (Lausanne) ; 8: 760236, 2021.

Article em En | MEDLINE | ID: mdl-34869463

Palavras-chave

Krabbe disease (globoid cell leukodystrophy); adeno-associated virus; galactocerebrosidase; gene therapy; leukodystrophies; psychosine

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Diagnostic_studies / Prognostic_studies Idioma: En Revista: Front Med (Lausanne) Ano de publicação: 2021 Tipo de documento: Article

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