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Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice
by
Sellier, P.
,
Vidal, P.
,
Bertin, B.
,
Gicquel, E.
,
Bertil‐Froidevaux, E.
,
Georger, C.
,
Wittenberghe, L.
,
Miranda, A.
,
Daniele, N.
,
Richard, I.
,
Gross, D. A.
,
Mingozzi, F.
,
Collaud, F.
,
Ronzitti, G.
Published in
Journal of inherited metabolic disease
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Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa −/− mice
by
Sellier, P.
,
Vidal, P.
,
Bertin, B.
,
Gicquel, E.
,
Bertil‐froidevaux, E
,
Georger, C.
,
van Wittenberghe, L.
,
Miranda, A.
,
Daniele, N.
,
Richard, Isabelle
,
Gross, D A
,
Mingozzi, F.
,
Collaud, F.
,
Ronzitti, G.
Published in
Journal of inherited metabolic disease
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Article
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Journal Of Inherited Metabolic Disease
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Life Sciences
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Aav
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Biochemistry, Molecular Biology
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Cardiac Muscle
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Clinical Trials
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Dual Promoter
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Endocrinology & Metabolism
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Expression Vectors
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Gene Therapy
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Genetics & Heredity
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Glycogen
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Immune Clearance
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Immune Response
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Immunogenicity
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Life Sciences & Biomedicine
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Liver
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Lysosomal Storage Disorders
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Medicine, Research & Experimental
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Molecular Biology
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Muscle Strength
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Wiley-Blackwell Journals (Backfile Content)
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Wiley Online Library - Autoholdings Journals
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Springer Link Archives
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Springerlink Contemporary
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