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
Biomedicines, Free Full-Text
Marta, with Niemann Pick C, fights for a cure with gene therapy
Haematologica, Volume 107, Issue 6 by Haematologica - Issuu
Marta, with Niemann Pick C, fights for a cure with gene therapy
Niemann Pick C Awareness Group
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
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Marta, with Niemann Pick C, fights for a cure with gene therapy
JBC Reviews: Journal of Biological Chemistry
Marta, with Niemann Pick C, fights for a cure with gene therapy
Selected Abstracts from the 12th Annual Meeting of the Clinical
Marta, with Niemann Pick C, fights for a cure with gene therapy
Gene Therapy for Niemann-Pick Type a Diseases Moves Forward
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
Hepatitis C Virus - Morphogenesis, Infection and Therapy
Marta, with Niemann Pick C, fights for a cure with gene therapy
Selected Abstracts from the 12th Annual Meeting of the Clinical
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
Gene therapy shows promise for treating Niemann-Pick disease type
Marta, with Niemann Pick C, fights for a cure with gene therapy
Marta – Columbus Children's Foundation
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