Batshaw, Mark L

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang, Y., Wang, L., Bell, P., McMenamin, D., He, Z., White, J., Yu, H., Xu, C., Morizono, H., Musunuru, K., et al. (2016) · Nature Biotechnology

Virus-based gene therapy researchCRISPR and Genetic Engineering

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Clinical research for rare disease: Opportunities, challenges, and solutions

Griggs, R. C., Batshaw, M., Dunkle, M., Gopal-Srivastava, R., Kaye, E., Krischer, J., Nguyen, T., Paulus, K., & Merkel, P. A. (2009) · Molecular Genetics and Metabolism

Statistical Methods in Clinical TrialsGenomics and Rare Diseases

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Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

Calcedo, R., Morizono, H., Wang, L., McCarter, R., He, J., Jones, D., Batshaw, M. L., & Wilson, J. M. (2011) · Clinical and Vaccine Immunology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 345

The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

Wang, L., Calcedo, R., Wang, H., Bell, P., Grant, R., Vandenberghe, L. H., Sanmiguel, J., Morizono, H., Batshaw, M. L., & Wilson, J. M. (2010) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

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