Liesbeth De Waele

Gain-of-function mutations in IFIH1 cause a spectrum of human disease phenotypes associated with upregulated type I interferon signaling

Gillian Rice, Yoandris del Toro Duany, Emma M. Jenkinson et al. · Nature Genetics · 2014 · 591 citations · Full text

Life Sciences, Genetics, Molecular Biology +16

Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

Thierry VandenDriessche, Lieven Thorrez, Abel Acosta‐Sanchez et al. · Journal of Thrombosis and Haemostasis · 2006 · 199 citations · Full text

Medicine, Hematology, Pharmaceutical Biotechnology +14

Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study

A. Chabanon, A. Seferian, Aurore Daron et al. · PLoS ONE · 2018 · 157 citations · Full text

Medicine, Neurology, Neuroscience +16

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

Janka Mátrai, Alessio Cantore, Cynthia C. Bartholomae et al. · Hepatology · 2011 · 136 citations · Full text

Life Sciences, Immunology, Molecular Genetics +13

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy

Michela Guglieri, Paula R. Clemens, Seth J. Perlman et al. · JAMA Neurology · 2022 · 130 citations · Full text

Pharmacy, Pharmacology, Pharmacotherapy +17