Smith, Alexander J.

Effect of gene therapy on visual function in Leber’s congenital amaurosis

Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 1,924

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

Bainbridge, J. W. B., Mehat, M. S., Sundaram, V., Robbie, S. J., Barker, S. E., Ripamonti, C., Georgiadis, A., Mowat, F. M., Beattie, S. G., Gardner, P. J., et al. (2015) · New England Journal of Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 743

Effective gene therapy with nonintegrating lentiviral vectors

Yáñez-Muñoz, R. J., Balaggan, K. S., MacNeil, A., Howe, S. J., Schmidt, M., Smith, A. J., Buch, P., MacLaren, R. E., Anderson, P. N., Barker, S. E., et al. (2006) · Nature Medicine

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 460

Correction of liver disease by hepatocyte transplantation in a mouse model of progressive familial intrahepatic cholestasis

De Vree, J. M. L., Ottenhoff, R., Bosma, P. J., Smith, A. J., Aten, J., & Oude Elferink, R. P. J. (2000) · Gastroenterology

Liver physiology and pathologyOrgan Transplantation Techniques and Outcomes

Cited by 186

Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8

Natkunarajah, M., Trittibach, P., McIntosh, J., Duran, Y., Barker, S. E., Smith, A. J., Nathwani, A. C., & Ali, R. R. (2008) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 138

Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

Georgiadis, A., Duran, Y., Ribeiro, J., Abelleira-Hervas, L., Robbie, S. J., Sünkel-Laing, B., Fourali, S., Gonzalez-Cordero, A., Cristante, E., Michaelides, M., et al. (2016) · Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 84

Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells

Gonzalez-Cordero, A., Goh, D., Kruczek, K., Naeem, A., Fernando, M., kleine Holthaus, S.-M., Takaaki, M., Blackford, S. J. I., Kloc, M., Agundez, L., et al. (2018) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 77

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice

Barker, S. E., Broderick, C. A., Robbie, S. J., Duran, Y., Natkunarajah, M., Buch, P., Balaggan, K. S., MacLaren, R. E., Bainbridge, J. W. B., Smith, A. J., et al. (2009) · The Journal of Gene Medicine

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 59

Stable RAAV-Mediated Transduction of Rod and Cone Photoreceptors in the Canine Retina

Bainbridge, J. W. B., Mistry, A., Schlichtenbrede, F. C., Smith, A., Broderick, C., De Alwis, M., Georgiadis, A., Taylor, P. M., Squires, M., Sethi, C., et al. (2003) · Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 58

Phase 1/2 AAV5-hRKp.RPGR (botaretigene sparoparvovec) gene therapy: safety and efficacy in RPGR-associated X-linked retinitis pigmentosa

MICHAELIDES, M., BESIRLI, C. G., YANG, Y., DE GUIMARAES, T. A. C., WONG, S. C., HUCKFELDT, R. M., COMANDER, J. I., SAHEL, J.-A., SHAH, S. M., TEE, J. J. L., et al. (2024) · American Journal of Ophthalmology

Retinal Development and DisordersVirus-based gene therapy research

Cited by 55

First-in-Human gene therapy trial of AAV8-hCARp.hCNGB3 in adults and children with CNGB3-associated achromatopsia

Michaelides, M., Hirji, N., Wong, S. C., Besirli, C. G., Zaman, S., Kumaran, N., Georgiadis, A., Smith, A. J., Ripamonti, C., Gottlob, I., et al. (2023) · American Journal of Ophthalmology

Retinal Development and DisordersVirus-based gene therapy research

Cited by 54

Antioxidant and lipid supplementation improve the development of photoreceptor outer segments in pluripotent stem cell-derived retinal organoids

West, E. L., Majumder, P., Naeem, A., Fernando, M., O'Hara-Wright, M., Lanning, E., Kloc, M., Ribeiro, J., Ovando-Roche, P., Shum, I. O., et al. (2022) · Stem Cell Reports

Retinal Development and DisordersPluripotent Stem Cells Research

Cited by 53