George, Lindsey A.

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Russell, S., Bennett, J., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., et al. (2017) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 1,952

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., Cuker, A., Sullivan, L. M., Majumdar, S., Teitel, J., et al. (2017) · New England Journal of Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 762

Current clinical applications of in vivo gene therapy with AAVs

Mendell, J. R., Al-Zaidy, S. A., Rodino-Klapac, L. R., Goodspeed, K., Gray, S. J., Kay, C. N., Boye, S. L., Boye, S. E., George, L. A., Salabarria, S., et al. (2021) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 743

Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia b

George, L. A., Ragni, M. V., Rasko, J. E. J., Raffini, L. J., Samelson-Jones, B. J., Ozelo, M., Hazbon, M., Runowski, A. R., Wellman, J. A., Wachtel, K., et al. (2020) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 213

Hemophilia gene therapy comes of age

George, L. A. (2017) · Hematology

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 58

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

High, K. A., George, L. A., Eyster, M. E., Sullivan, S. K., Ragni, M. V., Croteau, S. E., Samelson-Jones, B. J., Evans, M., Joseney-Antoine, M., Macdougall, A., et al. (2018) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 57

Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A

Nguyen, G. N., George, L. A., Siner, J. I., Davidson, R. J., Zander, C. B., Zheng, X. L., Arruda, V. R., Camire, R. M., & Sabatino, D. E. (2017) · Journal of Thrombosis and Haemostasis

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 50

Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity

Samelson-Jones, B. J., Finn, J. D., George, L. A., Camire, R. M., & Arruda, V. R. (2019) · JCI Insight

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 44