Locatelli, Franco and Corbacioglu, Selim and Hobbs, William and Frangoul, Haydar and Walters, Mark C. (2024) Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing. AMERICAN JOURNAL OF HEMATOLOGY, 99 (3). pp. 430-438. ISSN 0361-8609, 1096-8652
Full text not available from this repository. (Request a copy)Abstract
A growing number of gene therapy- and gene editing-based treatments for patients with sickle cell disease (SCD) are entering clinical trials. These treatments, designed to target the underlying cause of SCD, have the potential to provide functional cures, which until now were possible only through allogeneic hematopoietic stem cell transplant. However, as these novel approaches advance from early- to late-stage clinical trials, it is essential to identify physiologically and clinically relevant endpoints that can demonstrate the achievement of a functional cure for SCD. Here, we present an overview of the pathophysiology of SCD and current treatment options, review ongoing SCD clinical trials using gene therapy or gene editing approaches, and identify the most relevant endpoints for demonstrating the attainment of a functional cure for SCD.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | HEMATOLOGY 2020 GUIDELINES; PATIENT-REPORTED OUTCOMES; FETAL-HEMOGLOBIN; AMERICAN SOCIETY; UNITED-STATES; RISK-FACTORS; PAIN CRISES; L-GLUTAMINE; TRANSPLANTATION; ANEMIA |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 24 Apr 2024 06:01 |
| Last Modified: | 04 Mar 2025 10:05 |
| URI: | https://pred.uni-regensburg.de/id/eprint/60426 |
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