Flotte, Terence R.

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., et al. (2008) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 954

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

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Keeler, A. M., & Flotte, T. R. (2019) · Annual Review of Virology

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 322

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy

Lek, A., Wong, B., Keeler, A., Blackwood, M., Ma, K., Huang, S., Sylvia, K., Batista, A. R., Artinian, R., Kokoski, D., et al. (2023) · New England Journal of Medicine

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 319

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

Liu, P., Liang, S.-Q., Zheng, C., Mintzer, E., Zhao, Y. G., Ponnienselvan, K., Mir, A., Sontheimer, E. J., Gao, G., Flotte, T. R., et al. (2021) · Nature Communications

CRISPR and Genetic EngineeringRNA regulation and disease

Cited by 273

Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection

Jacobson, S. G., Acland, G. M., Aguirre, G. D., Aleman, T. S., Schwartz, S. B., Cideciyan, A. V., Zeiss, C. J., Komaromy, A. M., Kaushal, S., Roman, A. J., et al. (2006) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 224

In vivo model of adeno-associated virus vector persistence and rescue

Afione, S. A., Conrad, C. K., Kearns, W. G., Chunduru, S., Adams, R., Reynolds, T. C., Guggino, W. B., Cutting, G. R., Carter, B. J., & Flotte, T. R. (1996) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 223

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

Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

Wilson, J. M., & Flotte, T. R. (2020) · Human Gene Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 203

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

Gene Expression from Adeno-associated Virus Vectors in Airway Epithelial Cells

Flotte, T. R., Solow, R., Owens, R. A., Afione, S., Zeitlin, P. L., & Carter, B. J. (1992) · American Journal of Respiratory Cell and Molecular Biology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 167

Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer

Arjomandnejad, M., Dasgupta, I., Flotte, T. R., & Keeler, A. M. (2023) · BioDrugs

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 159

AAV gene therapy for Tay-Sachs disease

Flotte, T. R., Cataltepe, O., Puri, A., Batista, A. R., Moser, R., McKenna-Yasek, D., Douthwright, C., Gernoux, G., Blackwood, M., Mueller, C., et al. (2022) · Nature Medicine

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 143

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material

Lock, M., McGorray, S., Auricchio, A., Ayuso, E., Beecham, E. J., Blouin-Tavel, V., Bosch, F., Bose, M., Byrne, B. J., Caton, T., et al. (2010) · Human Gene Therapy

Virus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects

Cited by 131

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

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency

Calcedo, R., Somanathan, S., Qin, Q., Betts, M. R., Rech, A. J., Vonderheide, R. H., Mueller, C., Flotte, T. R., & Wilson, J. M. (2017) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 79

Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

Arjomandnejad, M., Sylvia, K., Blackwood, M., Nixon, T., Tang, Q., Muhuri, M., Gruntman, A. M., Gao, G., Flotte, T. R., & Keeler, A. M. (2021) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchCAR-T cell therapy research

Cited by 44

Training the next generation of biomedical investigators in glycosciences

Agre, P., Bertozzi, C., Bissell, M., Campbell, K. P., Cummings, R. D., Desai, U. R., Estes, M., Flotte, T., Fogleman, G., Gage, F., et al. (2016) · Journal of Clinical Investigation

Glycosylation and Glycoproteins ResearchHealth and Medical Research Impacts

Cited by 34

Methylphenidate Does Not Improve Cognitive Function in Healthy Sleep-Deprived Young Adults

Bray, C. L., Cahill, K. S., Oshier, J. T., Peden, C. S., Theriaque, D. W., Flotte, T. R., & Stacpoole, P. W. (2004) · Journal of Investigative Medicine

Neuroethics, Human Enhancement, Biomedical InnovationsSleep and Work-Related Fatigue

Cited by 29

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy

Lek, A., Keeler, A. M., & Flotte, T. R. (2023) · New England Journal of Medicine

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 12

The Promise of Gene Therapy For The Treatment of α-1 Antitrypsin Deficiency

Cruz, P. E., Mueller, C., & Flotte, T. R. (2007) · Pharmacogenomics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 10