Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
Cornell University · Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico · +4 more institutions
Abstract
in autologous CD34+ hematopoietic stem and progenitor cells (HSPCs).
-like genotype. CD34+ HSPCs were edited by means of CRISPR-Cas9 with a guide mRNA. Before the exa-cel infusion, patients underwent myeloablative conditioning with pharmacokinetically dose-adjusted busulfan. The primary end point was transfusion independence, defined as a weighted average hemoglobin level of 9 g per deciliter or higher without red-cell transfusion for at least 12 consecutive months. Total and fetal hemoglobin concentrations and safety were also assessed.
Citation impact
- FWCI
- 70.02
- Percentile
- 100%
- References
- 35
Authors
27- FLFranco LocatelliCorresponding
Cornell University, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Ospedale Maggiore, Düsseldorf University Hospital, Heinrich Heine University Düsseldorf, University College London
- PLPeter Lang
Cornell University, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Ospedale Maggiore, Düsseldorf University Hospital, Heinrich Heine University Düsseldorf, University College London
- DADonna A. Wall
Cornell University, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Ospedale Maggiore, Düsseldorf University Hospital, Heinrich Heine University Düsseldorf, University College London
- RMRoland Meisel
Cornell University, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Ospedale Maggiore, Düsseldorf University Hospital, Heinrich Heine University Düsseldorf, University College London
- SCSelim Corbacioglu
Cornell University, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Ospedale Maggiore, Düsseldorf University Hospital, Heinrich Heine University Düsseldorf, University College London
Topics & keywords
- Medicine
- Thalassemia
- Fetal hemoglobin
- Hemoglobin
- Internal medicine
- Gastroenterology
- Fetus
- Biology
- Good health and well-being