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Publications by M Vellard

Intrathecal Administration of AAV Vectors for the Treatment of Lysosomal Storage in the Brains of MPS I Mice

Gene Therapy
Molecular MedicineGeneticsMolecular Biology
2006English

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681. rDNA-AAV Vectors for the Treatment of Phenylketonuria

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Whole Genome Sequencing for Mutation Discovery in a Single Case of Lysosomal Storage Disease (MPS Type 1) in the Dog

Scientific Reports
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2020English

604. Intrathecal AAV9-Mediated Gene Delivery Corrects Lysosomal Storage Throughout the CNS in a Large Animal Model of Mucopolysaccharidosis Type I

Molecular Therapy
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The Molecular Basis of Lysosomal Storage Diseases and Their Treatment

Biochemical Society Transactions
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2000English

The Natural History of MPS I: Global Perspectives From the MPS I Registry

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Assaying the Stability and Inactivation of AAV Serotype 1 Vectors

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The Intrathecal Treatment of Tabes

BMJ
1914English

Growth Patterns for Untreated Individuals With MPS I: Report From the International MPS I Registry

American Journal of Medical Genetics, Part A
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221. The Utilization of Capsid-Optimized Adeno-Associated Virus (AAV) Vectors for Cancer Immunotherapy

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