Li, Chengwen

Engineering adeno-associated virus vectors for gene therapy

Li, C., & Samulski, R. J. (2020) · Nature Reviews Genetics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 1,175

Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

Rabinowitz, J. E., Rolling, F., Li, C., Conrath, H., Xiao, W., Xiao, X., & Samulski, R. J. (2002) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 762

Dystrophin Immunity in Duchenne's Muscular Dystrophy

Mendell, J. R., Campbell, K., Rodino-Klapac, L., Sahenk, Z., Shilling, C., Lewis, S., Bowles, D., Gray, S., Li, C., Galloway, G., et al. (2010) · New England Journal of Medicine

Muscle Physiology and DisordersVirus-based gene therapy research

Cited by 655

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

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

Chao, H., Liu, Y., Rabinowitz, J., Li, C., Samulski, R. J., & Walsh, C. E. (2000) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 366

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

Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

Nidetz, N. F., McGee, M. C., Tse, L. V., Li, C., Cong, L., Li, Y., & Huang, W. (2020) · Pharmacology & Therapeutics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 174

Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction

Shao, W., Earley, L. F., Chai, Z., Chen, X., Sun, J., He, T., Deng, M., Hirsch, M. L., Ting, J., Samulski, R. J., et al. (2018) · JCI Insight

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 119

Adeno-Associated Virus Serotype-Specific Inverted Terminal Repeat Sequence Role in Vector Transgene Expression

Earley, L. F., Conatser, L. M., Lue, V. M., Dobbins, A. L., Li, C., Hirsch, M. L., & Samulski, R. J. (2020) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 111

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

Blood-brain barrier shuttle peptides enhance AAV transduction in the brain after systemic administration

Zhang, X., He, T., Chai, Z., Samulski, R. J., & Li, C. (2018) · Biomaterials

Barrier Structure and Function StudiesVirus-based gene therapy research

Cited by 65

Oversized AAV Transductifon Is Mediated via a DNA-PKcs-independent, Rad51C-dependent Repair Pathway

Hirsch, M. L., Li, C., Bellon, I., Yin, C., Chavala, S., Pryadkina, M., Richard, I., & Samulski, R. J. (2013) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 62

Adeno-associated virus capsid antigen presentation is dependent on endosomal escape

Li, C., He, Y., Nicolson, S., Hirsch, M., Weinberg, M. S., Zhang, P., Kafri, T., & Samulski, R. J. (2013) · Journal of Clinical Investigation

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 61

Notable Reduction in Illegitimate Integration Mediated by a PPT-deleted, Nonintegrating Lentiviral Vector

Kantor, B., Bayer, M., Ma, H., Samulski, J., Li, C., McCown, T., & Kafri, T. (2011) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 56

Identification and Validation of Small Molecules That Enhance Recombinant Adeno-associated Virus Transduction following High-Throughput Screens

Nicolson, S. C., Li, C., Hirsch, M. L., Setola, V., & Samulski, R. J. (2016) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 48

Development of patient-specific AAV vectors after neutralizing antibody selection for enhanced muscle gene transfer

Li, C., Wu, S., Albright, B., Hirsch, M., Li, W., Tseng, Y.-S., Agbandje-McKenna, M., McPhee, S., Asokan, A., & Samulski, R. J. (2016) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 48

Cytotoxic-t-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo

Li, C., Hirsch, M., DiPrimio, N., Asokan, A., Goudy, K., Tisch, R., & Samulski, R. J. (2009) · Journal of Virology

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 42

Arsenic Trioxide Stabilizes Accumulations of Adeno-Associated Virus Virions at the Perinuclear Region, Increasing Transduction In Vitro and In Vivo

Mitchell, A. M., Li, C., & Samulski, R. J. (2013) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 41

Adeno-associated virus of a single-polarity DNA genome is capable of transduction in vivo

Zhou, X., Zeng, X., Fan, Z., Li, C., McCown, T., Samulski, R. J., & Xiao, X. (2008) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 41

Efficient capsid antigen presentation from adeno-associated virus empty virions in vivo

Pei, X., Earley, L. F., He, Y., Chen, X., Hall, N. E., Samulski, R. J., & Li, C. (2018) · Frontiers in Immunology

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 35

Quantitative 3D Tracing of Gene-delivery Viral Vectors in Human Cells and Animal Tissues

Xiao, P.-J., Li, C., Neumann, A., & Samulski, R. J. (2012) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 33

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