McCarty, Douglas M.

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

McCarty, D. M., Fu, H., Monahan, P. E., Toulson, C. E., Naik, P., & Samulski, R. J. (2003) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 547

Self-complementary AAV vectors; advances and applications

McCarty, D. M. (2008) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 515

Integration of Adeno-Associated Virus (AAV) and Recombinant AAV Vectors

McCarty, D. M., Young, S. M., & Samulski, R. J. (2004) · Annual Review of Genetics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 450

AAV hybrid serotypes: improved vectors for gene delivery

Choi, V., McCarty, D., & Samulski, R. (2005) · Current Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 191

Host Cell DNA Repair Pathways in Adeno-Associated Viral Genome Processing

Choi, V. W., McCarty, D. M., & Samulski, R. J. (2006) · Journal of Virology

Virus-based gene therapy researchDNA Repair Mechanisms

Cited by 144

Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

McCarty, D. M., DiRosario, J., Gulaid, K., Muenzer, J., & Fu, H. (2009) · Gene Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 105

Biodistribution and Tolerability of AAV-PHP.B-CBh- SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles

Palazzi, X., Pardo, I. D., Sirivelu, M. P., Newman, L., Kumpf, S. W., Qian, J., Franks, T., Lopes, S., Liu, J., Monarski, L., et al. (2022) · Human Gene Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 60

Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy

Fu, H., Meadows, A. S., Pineda, R. J., Kunkler, K. L., Truxal, K. V., McBride, K. L., Flanigan, K. M., & McCarty, D. M. (2017) · Human Gene Therapy Clinical Development

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 44

Effective depletion of pre-existing anti-AAV antibodies requires broad immune targeting

Velazquez, V. M., Meadows, A. S., Pineda, R. J., Camboni, M., McCarty, D. M., & Fu, H. (2017) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 43

Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice

Duncan, F. J., Naughton, B. J., Zaraspe, K., Murrey, D. A., Meadows, A. S., Clark, K. R., Newsom, D. E., White, P., Fu, H., & McCarty, D. M. (2015) · Molecular Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 25