Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing
Titel:
Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing
Auteur:
Esrick, Erica B. Achebe, Maureen Armant, Myriam Bartolucci, Pablo Ciuculescu, Marioara Felicia Daley, Heather Dansereau, Colleen Di Caprio, Giuseppe Goncalves, Bronner Hebert, Nicolas Heeney, Matthew M Higgins, John M. Lehmann, Leslie E. Manis, John P Negre, Olivier Nikiforow, Sarah Schonbrun, Ethan Wood, David K. Williams, David A.