Τίτλος:
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Γλώσσες Τεκμηρίου:
Αγγλικά
Περίληψη:
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are the most common inherited hematologic disorders, affecting approximately 60,000 and 300,000 patients worldwide, respectively. Current therapies, including red blood cell (RBC) transfusion and iron chelation in TDT and transfusion, pain management, and hydroxyurea in SCD, help to manage the disorders but do not address the underlying cause. Drug therapies, such as crizanlizumab and luspatercept, have also helped to reduce the need for transfusion in TDT patients and the incidence of vaso-occlusive episodes in SCD patients. Allogeneic bone marrow transplantationmay be a curative option, but finding an appropriate donor is difficult. An association has been observed between elevated levels of fetal hemoglobin and improved morbidity and mortality in these patients. Downregulating BCL11A, a transcription factor that blocks fetal hemoglobin in erythroid cells, may help to increase fetal hemoglobin levels and improve outcomes. Using the CRISPR-Cas9 gene-editing technique, CTX001, an investigational drug, was infused in 2 patients. This article describes the results of infusing CTX001 in 1 patient with TDT and another with SCD. © 2021 Wolters Kluwer Health, Inc. All rights reserved.
Συγγραφείς:
Frangoul, H.
Altshuler, D.
Cappellini, M.D.
Chen, Y.-S.
Domm, J.
Eustace, B.K.
Foell, J.
De La Fuente, J.
Grupp, S.
Handgretinger, R.
Ho, T.W.
Kattamis, A.
Kernytsky, A.
Lekstrom-Himes, J.
Li, A.M.
Locatelli, F.
Mapara, M.Y.
De Montalembert, M.
Rondelli, D.
Sharma, A.
Sheth, S.
Soni, S.
Steinberg, M.H.
Wall, D.
Yen, A.
Corbacioglu, S.
Περιοδικό:
Obstetrical and Gynecological Survey
Εκδότης:
Lippincott Williams and Wilkins
Λέξεις-κλειδιά:
crizanlizumab; exagamglogene autotemcel; hemoglobin; hemoglobin S; hydroxyurea; luspatercept; transcription factor; transcription factor BCL11A; unclassified drug, abdominal pain; allele; allogenic bone marrow transplantation; analgesia; beta thalassemia; bone marrow; CRISPR-CAS9 system; donor selection; down regulation; erythrocyte transfusion; erythroid cell; follow up; gallstone; gene editing; hemoglobin blood level; human; iron chelation; liver venoocclusive disease; morbidity; mortality; neutropenia; pneumonia; Review; sepsis; sickle cell anemia; sickle cell crisis; treatment outcome
DOI:
10.1097/01.ogx.0000754392.61396.79
