Marta, with Niemann Pick C, fights for a cure with gene therapy

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Marta is a four and a half year old girl with Niemann Pick C. This is a rare neurodegenerative genetic disease
Marta, with Niemann Pick C, fights for a cure with gene therapy
Lysosomal cholesterol activates mTORC1 via an SLC38A9–Niemann-Pick
Marta, with Niemann Pick C, fights for a cure with gene therapy
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Marta, with Niemann Pick C, fights for a cure with gene therapy
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Marta, with Niemann Pick C, fights for a cure with gene therapy
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Marta, with Niemann Pick C, fights for a cure with gene therapy
High sphingomyelin levels induce lysosomal damage and autophagy
Marta, with Niemann Pick C, fights for a cure with gene therapy
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Marta, with Niemann Pick C, fights for a cure with gene therapy
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Marta, with Niemann Pick C, fights for a cure with gene therapy
Lysosomal cholesterol activates mTORC1 via an SLC38A9–Niemann-Pick
Marta, with Niemann Pick C, fights for a cure with gene therapy
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Marta, with Niemann Pick C, fights for a cure with gene therapy
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Marta, with Niemann Pick C, fights for a cure with gene therapy
Marta, with Niemann Pick C, fights for a cure with gene therapy
Marta, with Niemann Pick C, fights for a cure with gene therapy
Neonatal brain-directed gene therapy rescues a mouse model of
Marta, with Niemann Pick C, fights for a cure with gene therapy
Marta, with Niemann Pick C, fights for a cure with gene therapy
Marta, with Niemann Pick C, fights for a cure with gene therapy
High sphingomyelin levels induce lysosomal damage and autophagy
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