Monahan, Paul E.

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

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Bowles, D. E., McPhee, S. W., Li, C., Gray, S. J., Samulski, J. J., Camp, A. S., Li, J., Wang, B., Monahan, P. E., Rabinowitz, J. E., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 400

Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

Li, C., Narkbunnam, N., Samulski, R. J., Asokan, A., Hu, G., Jacobson, L. J., Manco-Johnson, M. J., Monahan, P. E., & The Joint Outcome Study Investigators (2012) · Gene Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 225

BAX 335 hemophilia b gene therapy clinical trial results: Potential impact of CpG sequences on gene expression

Konkle, B. A., Walsh, C. E., Escobar, M. A., Josephson, N. C., Young, G., von Drygalski, A., McPhee, S. W. J., Samulski, R. J., Bilic, I., de la Rosa, M., et al. (2021) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 189

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose

Wu, Z., Sun, J., Zhang, T., Yin, C., Yin, F., Van Dyke, T., Samulski, R. J., & Monahan, P. E. (2008) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 153

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

Monahan, P. E., Lothrop, C. D., Sun, J., Hirsch, M. L., Kafri, T., Kantor, B., Sarkar, R., Tillson, D. M., Elia, J. R., & Samulski, R. J. (2010) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 125

Men with severe hemophilia in the United States: birth cohort analysis of a large national database

Mazepa, M. A., Monahan, P. E., Baker, J. R., Riske, B. K., & Soucie, J. M. (2016) · Blood

Hemophilia Treatment and ResearchPlatelet Disorders and Treatments

Cited by 117

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

Monahan, P. E., Sun, J., Gui, T., Hu, G., Hannah, W. B., Wichlan, D. G., Wu, Z., Grieger, J. C., Li, C., Suwanmanee, T., et al. (2015) · Human Gene Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 108

Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles

Li, C., Diprimio, N., Bowles, D. E., Hirsch, M. L., Monahan, P. E., Asokan, A., Rabinowitz, J., Agbandje-McKenna, M., & Samulski, R. J. (2012) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 96

Cellular immune response to cryptic epitopes during therapeutic gene transfer

Li, C., Goudy, K., Hirsch, M., Asokan, A., Fan, Y., Alexander, J., Sun, J., Monahan, P., Seiber, D., Sidney, J., et al. (2009) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 79

Gene therapy in an era of emerging treatment options for hemophilia B

Monahan, P. E. (2015) · Journal of Thrombosis and Haemostasis

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 31

An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

Sun, J., Shao, W., Chen, X., Merricks, E. P., Wimsey, L., Abajas, Y. L., Niemeyer, G. P., Lothrop, C. D., Monahan, P. E., Samulski, R. J., et al. (2018) · Molecular Therapy - Methods & Clinical Development

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 30

Hemophilia A and B mice, but not VWF−/−mice, display bone defects in congenital development and remodeling after injury

Taves, S., Sun, J., Livingston, E. W., Chen, X., Amiaud, J., Brion, R., Hannah, W. B., Bateman, T. A., Heymann, D., & Monahan, P. E. (2019) · Scientific Reports

Hemophilia Treatment and ResearchBone Metabolism and Diseases

Cited by 26