Conlon, Thomas J.

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

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Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo

Pacak, C. A., Mah, C. S., Thattaliyath, B. D., Conlon, T. J., Lewis, M. A., Cloutier, D. E., Zolotukhin, I., Tarantal, A. F., & Byrne, B. J. (2006) · Circulation Research

Virus-based gene therapy researchRNA Interference and Gene Delivery

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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

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Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

Cideciyan, A. V., Hauswirth, W. W., Aleman, T. S., Kaushal, S., Schwartz, S. B., Boye, S. L., Windsor, E. A. M., Conlon, T. J., Sumaroka, A., Pang, J.-J., et al. (2009) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

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Sustained alpha‐sarcoglycan gene expression after gene transfer in limb‐girdle muscular dystrophy, type 2D

Mendell, J. R., Rodino‐Klapac, L. R., Rosales, X. Q., Coley, B. D., Galloway, G., Lewis, S., Malik, V., Shilling, C., Byrne, B. J., Conlon, T., et al. (2010) · Annals of Neurology

Virus-based gene therapy researchMuscle Physiology and Disorders

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Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial

Ghazi, N. G., Abboud, E. B., Nowilaty, S. R., Alkuraya, H., Alhommadi, A., Cai, H., Hou, R., Deng, W.-T., Boye, S. L., Almaghamsi, A., et al. (2016) · Human Genetics

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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

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Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

Smith, B. K., Collins, S. W., Conlon, T. J., Mah, C. S., Lawson, L. A., Martin, A. D., Fuller, D. D., Cleaver, B. D., Clément, N., Phillips, D., et al. (2013) · Human Gene Therapy

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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

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Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV- hGAA ) Gene Therapy in Children Affected by Pompe Disease

Corti, M., Liberati, C., Smith, B. K., Lawson, L. A., Tuna, I. S., Conlon, T. J., Coleman, K. E., Islam, S., Herzog, R. W., Fuller, D. D., et al. (2017) · Human Gene Therapy Clinical Development

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The Human Rhodopsin Kinase Promoter in an AAV5 Vector Confers Rod- and Cone-Specific Expression in the Primate Retina

Boye, S. E., Alexander, J. J., Boye, S. L., Witherspoon, C. D., Sandefer, K. J., Conlon, T. J., Erger, K., Sun, J., Ryals, R., Chiodo, V. A., et al. (2012) · Human Gene Therapy

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Pompe disease gene therapy

Byrne, B. J., Falk, D. J., Pacak, C. A., Nayak, S., Herzog, R. W., Elder, M. E., Collins, S. W., Conlon, T. J., Clement, N., Cleaver, B. D., et al. (2011) · Human Molecular Genetics

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LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile

Koilkonda, R., Yu, H., Talla, V., Porciatti, V., Feuer, W. J., Hauswirth, W. W., Chiodo, V., Erger, K. E., Boye, S. L., Lewin, A. S., et al. (2014) · Investigative Ophthalmology & Visual Science

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Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs

Boyd, R. F., Boye, S. L., Conlon, T. J., Erger, K. E., Sledge, D. G., Langohr, I. M., Hauswirth, W. W., Komáromy, A. M., Boye, S. E., Petersen-Jones, S. M., et al. (2016) · Gene Therapy

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Toxicology and pharmacology of an AAV vector expressing codon-optimized RPGR in RPGR-deficient Rd9 mice

Song, C., Conlon, T. J., Deng, W.-T., Coleman, K. E., Zhu, P., Plummer, C., Mandapati, S., Van Hoosear, M., Green, K. B., Sonnentag, P., et al. (2018) · Human Gene Therapy Clinical Development

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