Schmidt, Manfred

Lentiviral Hematopoietic Cell Gene Therapy for X-Linked Adrenoleukodystrophy

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Bougnères, P., Schmidt, M., Von Kalle, C., Fischer, A., Cavazzana-Calvo, M., & Aubourg, P. (2012) · Methods in Enzymology

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 123

Extensive Methylation of Promoter Sequences Silences Lentiviral Transgene Expression During Stem Cell Differentiation In Vivo

Herbst, F., Ball, C. R., Tuorto, F., Nowrouzi, A., Wang, W., Zavidij, O., Dieter, S. M., Fessler, S., van der Hoeven, F., Kloz, U., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchEpigenetics and DNA Methylation

Cited by 104

AAV vector-mediated in vivo reprogramming into pluripotency

Senís, E., Mosteiro, L., Wilkening, S., Wiedtke, E., Nowrouzi, A., Afzal, S., Fronza, R., Landerer, H., Abad, M., Niopek, D., et al. (2018) · Nature Communications

Pluripotent Stem Cells ResearchVirus-based gene therapy research

Cited by 64

Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Bougnères, P., Hacein-Bey-Abina, S., Labik, I., Adamsbaum, C., Castaignède, C., Bellesme, C., & Schmidt, M. (2021) · Human Gene Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 45

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Kanter, J., Tisdale, J. F., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Huang, W., Ribeil, J.-A., et al. (2019) · Blood

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 21