DROSHA Knockout Leads to Enhancement of Viral Titers for Vectors Encoding miRNA-Adapted shRNAs

por Park, Hee Ho, Triboulet, Robinson, Bentler, Martin, Guda, Swaroopa, Du, Peng, Xu, Haiming, Gregory, Richard I., Brendel, Christian, Williams, David A.
Publicado 2018

Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease

por Dogan, Yildirim, Barese, Cecilia N., Schindler, Jeffrey W., Yoon, John K., Unnisa, Zeenath, Guda, Swaroopa, Jacobs, Mary E., Oborski, Christine, Maiwald, Tim, Clarke, Diana L., Schambach, Axel, Pfeifer, Richard, Harper, Claudia, Mason, Chris, van Til, Niek P.
Publicado 2022

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