Nichols, Timothy C.

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

Snyder, R. O., Miao, C., Meuse, L., Tubb, J., Donahue, B. A., Lin, H.-F., Stafford, D. W., Patel, S., Thompson, A. R., Nichols, T., et al. (1999) · Nature Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 409

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

Mount, J. D., Herzog, R. W., Tillson, D. M., Goodman, S. A., Robinson, N., McCleland, M. L., Bellinger, D., Nichols, T. C., Arruda, V. R., Lothrop, C. D., et al. (2002) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 339

Sustained correction of disease in naive and AAV2-pretreated hemophilia b dogs: AAV2/8-mediated, liver-directed gene therapy

Wang, L., Calcedo, R., Nichols, T. C., Bellinger, D. A., Dillow, A., Verma, I. M., & Wilson, J. M. (2005) · Blood

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 173

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Cantore, A., Ranzani, M., Bartholomae, C. C., Volpin, M., Valle, P. D., Sanvito, F., Sergi, L. S., Gallina, P., Benedicenti, F., Bellinger, D., et al. (2015) · Science Translational Medicine

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 158

Eradication of neutralizing antibodies to factor VIII in canine hemophilia a after liver gene therapy

Finn, J. D., Ozelo, M. C., Sabatino, D. E., Franck, H. W. G., Merricks, E. P., Crudele, J. M., Zhou, S., Kazazian, H. H., Lillicrap, D., Nichols, T. C., et al. (2010) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 157

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

Herzog, R. W., Fields, P. A., Arruda, V. R., Brubaker, J. O., Armstrong, E., McClintock, D., Bellinger, D. A., Couto, L. B., Nichols, T. C., & High, K. A. (2002) · Human Gene Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 147

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

Mátrai, J., Cantore, A., Bartholomae, C. C., Annoni, A., Wang, W., Acosta-Sanchez, A., Samara-Kuko, E., De Waele, L., Ma, L., Genovese, P., et al. (2011) · Hepatology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 139

Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A

Du, L. M., Nurden, P., Nurden, A. T., Nichols, T. C., Bellinger, D. A., Jensen, E. S., Haberichter, S. L., Merricks, E., Raymer, R. A., Fang, J., et al. (2013) · Nature Communications

Hemophilia Treatment and ResearchPlatelet Disorders and Treatments

Cited by 119

Evolutionary Insights Into Coagulation Factor IX Padua and Other High-Specific-Activity Variants

Samelson-Jones, B. J., Finn, J. D., Raffini, L. J., Merricks, E. P., Camire, R. M., Nichols, T. C., & Arruda, V. R. (2021) · Blood Advances

Hemophilia Treatment and ResearchBlood Coagulation and Thrombosis Mechanisms

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

Superior human hepatocyte transduction with adeno-associated virus vector serotype 7

Shao, W., Pei, X., Cui, C., Askew, C., Dobbins, A., Chen, X., Abajas, Y. L., Gerber, D. A., Samulski, R. J., Nichols, T. C., et al. (2019) · Gene Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 22