Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

MicroRNA maturation: stepwise processing and subcellular localization

Yoontae Lee · The EMBO Journal · 2002 · 2.4K citations · Full text

Microrna Maturation, Microrna Detection, Small Rna +4

Position-independent, high-level expression of the human β-globin gene in transgenic mice

Frank Grosveld, Greet Blom van Assendelft, David R. Greaves et al. · Cell · 1987 · 1.9K citations · Full text

Transcriptional Regulation, Protein Expression, High-level Expression +7

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Salima Hacein‐Bey‐Abina, Alexandrine Garrigue, Gary P. Wang et al. · Journal of Clinical Investigation · 2008 · 1.8K citations · Full text

Viral Replication, Mixed-phenotype Acute Leukemia, Immunology +23

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Nathalie Cartier, Salima Hacein‐Bey‐Abina, Cynthia C. Bartholomae et al. · Science · 2009 · 1.5K citations · Full text

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1

Marion Ott, Manfred Schmidt, Kerstin Schwarzwaelder et al. · Nature Medicine · 2006 · 1.2K citations

Gene Therapies, Autoimmune Disease, Novel Therapy +10