Daniela Abriss

Post-Transcriptional Genetic Silencing of <i>BCL11A</i> to Treat Sickle Cell Disease

Erica B. Esrick, Leslie Lehmann, Alessandra Biffi et al. · New England Journal of Medicine · 2020 · 407 citations · Full text

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy

Christian Brendel, Olivier Nègre, Michael Rothe et al. · Molecular Therapy — Methods & Clinical Development · 2020 · 67 citations · Full text

Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A

Stefanie Roth, Jörg Schüttrumpf, Peter Milanov et al. · PLoS ONE · 2012 · 45 citations · Full text

Protein Secretion, Molecular Biology, Oxidative Stress +16

Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for <i>Ex Vivo</i> Gene Therapy of X-Linked Chronic Granulomatous Disease

Christian Brendel, Michael Rothe, Giorgia Santilli et al. · Human Gene Therapy Clinical Development · 2018 · 36 citations

Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice

Peter Milanov, Lacramioara Ivanciu, Daniela Abriss et al. · Blood · 2011 · 31 citations · Full text