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

por Roth, Stefanie D., Schüttrumpf, Jörg, Milanov, Peter, Abriss, Daniela, Ungerer, Christopher, Quade-Lyssy, Patricia, Simpson, Jeremy C., Pepperkok, Rainer, Seifried, Erhard, Tonn, Torsten
Publicado 2012

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

por Brendel, Christian, Negre, Olivier, Rothe, Michael, Guda, Swaroopa, Parsons, Geoff, Harris, Chad, McGuinness, Meaghan, Abriss, Daniela, Tsytsykova, Alla, Klatt, Denise, Bentler, Martin, Pellin, Danilo, Christiansen, Lauryn, Schambach, Axel, Manis, John, Trebeden-Negre, Helene, Bonner, Melissa, Esrick, Erica, Veres, Gabor, Armant, Myriam, Williams, David A.
Publicado 2020