Humphries, Margaret

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α 1 -Antitrypsin: Interim Results

Flotte, T. R., Trapnell, B. C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A. T., Sandhaus, R. A., McElvaney, N. G., et al. (2011) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 335

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Brantly, M. L., Chulay, J. D., Wang, L., Mueller, C., Humphries, M., Spencer, L. T., Rouhani, F., Conlon, T. J., Calcedo, R., Betts, M. R., et al. (2009) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 327

Results at 2 Years after gene therapy for RPE65-deficient leber congenital amaurosis and severe early-childhood–onset retinal dystrophy

Weleber, R. G., Pennesi, M. E., Wilson, D. J., Kaushal, S., Erker, L. R., Jensen, L., McBride, M. T., Flotte, T. R., Humphries, M., Calcedo, R., et al. (2016) · Ophthalmology

Retinal Development and DisordersVirus-based gene therapy research

Cited by 218

Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

Mueller, C., Chulay, J. D., Trapnell, B. C., Humphries, M., Carey, B., Sandhaus, R. A., McElvaney, N. G., Messina, L., Tang, Q., Rouhani, F. N., et al. (2013) · Journal of Clinical Investigation

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 169

5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency

Mueller, C., Gernoux, G., Gruntman, A. M., Borel, F., Reeves, E. P., Calcedo, R., Rouhani, F. N., Yachnis, A., Humphries, M., Campbell-Thompson, M., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 112