Year: 2026
Type: article
Abstract: potential.Furthermore, a recent study using CRISPR/Cas9 to introduce cisacting elements for KLF1 and GATA1/ TAL1 cobinding motifs in the HBD promoter, produced gene activation in SCD mice.9 From a clinical perspective, modest increases in HbF of >8.6% was associated with improved long-term survival in the Cooperative Study of Sickle Cell Disease. 10 This suggests that pharmacological induction of HBD via the IFNγ/JAK/STAT axis may represent a novel therapeutic approach.However, prolonged IFN-γ exposure suppresses erythropoiesis and causes anemia; to address this limitation, the authors proposed strategies such as a short-course/ intermittent ("pulsed") dosing or combining IFN-γ with erythropoietin to mitigate toxicity.How will HbA 2 induction by IFN-γ compare to the efficacy, safety, and practicality of established oral hydroxyurea treatment or other small molecules under development as HbF inducers?Additional studies where hydroxyurea is combined with IFN-γ, will shed light on this important question.
Source: Blood
Authors: Ghassan Zammar, Chan Y. Cheah
Language: English
Subfield: Pathology and Forensic Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: bronze
OpenAlex ID: W7133645192