Timmers, Adrian M.

A Muscleblind Knockout Model for Myotonic Dystrophy

Kanadia, R. N., Johnstone, K. A., Mankodi, A., Lungu, C., Thornton, C. A., Esson, D., Timmers, A. M., Hauswirth, W. W., & Swanson, M. S. (2003) · Science

Genetic Neurodegenerative DiseasesRNA Research and Splicing

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Ocular Inflammatory Response to Intravitreal Injection of Adeno-Associated Virus Vector: Relative Contribution of Genome and Capsid

Timmers, A. M., Newmark, J. A., Turunen, H. T., Farivar, T., Liu, J., Song, C., Ye, G.-J., Pennock, S., Gaskin, C., Knop, D. R., et al. (2020) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

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Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

Dufour, V. L., Cideciyan, A. V., Ye, G.-J., Song, C., Timmers, A., Habecker, P. L., Pan, W., Weinstein, N. M., Swider, M., Durham, A. C., et al. (2020) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

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Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

Song, C., Dufour, V. L., Cideciyan, A. V., Ye, G.-J., Swider, M., Newmark, J. A., Timmers, A. M., Robinson, P. M., Knop, D. R., Chulay, J. D., et al. (2020) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

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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

Retinal Development and DisordersVirus-based gene therapy research

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