Hauswirth, William W.

Gene therapy restores vision in a canine model of childhood blindness

Acland, G. M., Aguirre, G. D., Ray, J., Zhang, Q., Aleman, T. S., Cideciyan, A. V., Pearce-Kelling, S. E., Anand, V., Zeng, Y., Maguire, A. M., et al. (2001) · Nature Genetics

Retinal Development and DisordersVirus-based gene therapy research

Cited by 1,202

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

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

Cited by 743

DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration

Kaneko, H., Dridi, S., Tarallo, V., Gelfand, B. D., Fowler, B. J., Cho, W. G., Kleinman, M. E., Ponicsan, S. L., Hauswirth, W. W., Chiodo, V. A., et al. (2011) · Nature

Retinal Diseases and TreatmentsRetinal Development and Disorders

Cited by 601

DICER1 loss and Alu RNA induce age-related macular degeneration via the NLRP3 inflammasome and MyD88

Tarallo, V., Hirano, Y., Gelfand, B. D., Dridi, S., Kerur, N., Kim, Y., Cho, W. G., Kaneko, H., Fowler, B. J., Bogdanovich, S., et al. (2012) · Cell

Inflammasome and immune disordersRetinal Diseases and Treatments

Cited by 592

High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors

Petrs-Silva, H., Dinculescu, A., Li, Q., Min, S.-H., Chiodo, V., Pang, J.-J., Zhong, L., Zolotukhin, S., Srivastava, A., Lewin, A. S., et al. (2009) · Molecular Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 398

Gene therapy with brain-derived neurotrophic factor as a protection: Retinal ganglion cells in a rat glaucoma model

Martin, K. R. G., Quigley, H. A., Zack, D. J., Levkovitch-Verbin, H., Kielczewski, J., Valenta, D., Baumrind, L., Pease, M. E., Klein, R. L., & Hauswirth, W. W. (2003) · Investigative Opthalmology & Visual Science

Glaucoma and retinal disordersVirus-based gene therapy research

Cited by 362

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

Cited by 312

Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

Akil, O., Dyka, F., Calvet, C., Emptoz, A., Lahlou, G., Nouaille, S., Boutet de Monvel, J., Hardelin, J.-P., Hauswirth, W. W., Avan, P., et al. (2019) · Proceedings of the National Academy of Sciences

Hearing, Cochlea, Tinnitus, GeneticsVirus-based gene therapy research

Cited by 300

Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina

Petrs-Silva, H., Dinculescu, A., Li, Q., Deng, W.-T., Pang, J.-J., Min, S.-H., Chiodo, V., Neeley, A. W., Govindasamy, L., Bennett, A., et al. (2011) · Molecular Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 267

A comprehensive review of retinal gene therapy

Boye, S. E., Boye, S. L., Lewin, A. S., & Hauswirth, W. W. (2013) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 262

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

Beltran, W. A., Cideciyan, A. V., Lewin, A. S., Iwabe, S., Khanna, H., Sumaroka, A., Chiodo, V. A., Fajardo, D. S., Román, A. J., Deng, W.-T., et al. (2012) · Proceedings of the National Academy of Sciences

Retinal Development and DisordersVirus-based gene therapy research

Cited by 260

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

Retinal Development and DisordersVirus-based gene therapy research

Cited by 243

SOD2 knockdown mouse model of early AMD

Justilien, V., Pang, J.-J., Renganathan, K., Zhan, X., Crabb, J. W., Kim, S. R., Sparrow, J. R., Hauswirth, W. W., & Lewin, A. S. (2007) · Investigative Opthalmology & Visual Science

Retinal Diseases and TreatmentsRetinal Development and Disorders

Cited by 234

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

Effect of CNTF on retinal ganglion cell survival in experimental glaucoma

Pease, M. E., Zack, D. J., Berlinicke, C., Bloom, K., Cone, F., Wang, Y., Klein, R. L., Hauswirth, W. W., & Quigley, H. A. (2009) · Investigative Opthalmology & Visual Science

Glaucoma and retinal disordersNerve injury and regeneration

Cited by 222

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

Gene Therapy for Leber Hereditary Optic Neuropathy

Guy, J., Feuer, W. J., Davis, J. L., Porciatti, V., Gonzalez, P. J., Koilkonda, R. D., Yuan, H., Hauswirth, W. W., & Lam, B. L. (2017) · Ophthalmology

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 204

Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy

LaVail, M. M., Yasumura, D., Matthes, M. T., Drenser, K. A., Flannery, J. G., Lewin, A. S., & Hauswirth, W. W. (2000) · Proceedings of the National Academy of Sciences

Retinal Development and DisordersRNA Interference and Gene Delivery

Cited by 203

Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors

Kay, C. N., Ryals, R. C., Aslanidi, G. V., Min, S. H., Ruan, Q., Sun, J., Dyka, F. M., Kasuga, D., Ayala, A. E., Van Vliet, K., et al. (2013) · PLoS ONE

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 168

Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector

Cideciyan, A. V., Sudharsan, R., Dufour, V. L., Massengill, M. T., Iwabe, S., Swider, M., Lisi, B., Sumaroka, A., Marinho, L. F., Appelbaum, T., et al. (2018) · Proceedings of the National Academy of Sciences

Retinal Development and DisordersVirus-based gene therapy research

Cited by 147

Pharmacological and rAAV Gene Therapy Rescue of Visual Functions in a Blind Mouse Model of Leber Congenital Amaurosis

Batten, M. L., Imanishi, Y., Tu, D. C., Doan, T., Zhu, L., Pang, J., Glushakova, L., Moise, A. R., Baehr, W., Van Gelder, R. N., et al. (2005) · PLoS Medicine

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 134

Dual Adeno-Associated Virus Vectors Result in Efficient In Vitro and In Vivo Expression of an Oversized Gene, MYO7A

Dyka, F. M., Boye, S. L., Chiodo, V. A., Hauswirth, W. W., & Boye, S. E. (2014) · Human Gene Therapy Methods

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 128

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

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 117