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Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model

Molecular Therapy - United States
doi 10.1016/j.ymthe.2017.01.009
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Abstract

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Categories
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
Date

April 1, 2017

Authors
Heather L. Gray-EdwardsDebra S. RegierJamie L. ShirleyAshley N. RandleNouha SalibiSarah E. ThomasYvonne L. LatourJean JohnstonGretchen GolasAnnie S. MaguireAmanda R. TaylorDonald C. SorjonenVictoria J. McCurdyPeter W. ChristophersonAllison M. BradburyRonald J. BeyersAime K. JohnsonBrandon L. BrunsonNancy R. CoxHenry J. BakerThomas S. DenneyMiguel Sena-EstevesCynthia J. TifftDouglas R. Martin
Publisher

Elsevier BV

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