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Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects |
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Titel: |
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects |
Auteur: |
Colella, Pasqualina Sellier, Pauline Gomez, Manuel J. Biferi, Maria G. Tanniou, Guillaume Guerchet, Nicolas Cohen-Tannoudji, Mathilde Moya-Nilges, Maryse van Wittenberghe, Laetitia Daniele, Natalie Gjata, Bernard Krijnse-Locker, Jacomina Collaud, Fanny Simon-Sola, Marcelo Charles, Severine Cagin, Umut Mingozzi, Federico |
Verschenen in: |
EBioMedicine |
Paginering: |
Jaargang 61 () nr. C pagina's p. |
Jaar: |
2020 |
Inhoud: |
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Uitgever: |
The Authors |
Bronbestand: |
Elektronische Wetenschappelijke Tijdschriften |
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