Thakar, Himal L.

Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy

Duncan, C. N., Bledsoe, J. R., Grzywacz, B., Beckman, A., Bonner, M., Eichler, F. S., Kühl, J.-S., Harris, M. H., Slauson, S., Colvin, R. A., et al. (2024) · New England Journal of Medicine

Virus-based gene therapy researchLysosomal Storage Disorders Research

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Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

Eichler, F., Duncan, C. N., Musolino, P. L., Lund, T. C., Gupta, A. O., De Oliveira, S., Thrasher, A. J., Aubourg, P., Kühl, J.-S., Loes, D. J., et al. (2024) · New England Journal of Medicine

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 107

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Thompson, A. A., Olson, T. S., Walters, M. C., Porter, J. B., Schneiderman, J., Hongeng, S., Kulozik, A., Cavazzana, M., Sauer, M. G., Thrasher, A. J., et al. (2023) · Blood

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 6