Combined approaches for increasing fetal hemoglobin (HbF) and de novo production of adult hemoglobin (HbA) in erythroid cells from β-thalassemia patients …

A Finotti, R Gambari - Frontiers in Genome Editing, 2023 - frontiersin.org
Genome editing (GE) is one of the most efficient and useful molecular approaches to correct
the effects of gene mutations in hereditary monogenetic diseases, including β-thalassemia …

A comprehensive overview of CRISPR/Cas 9 technology and application thereof in drug discovery

A Khurana, N Sayed, V Singh, I Khurana… - Journal of Cellular …, 2022 - Wiley Online Library
Abstract Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)‐Cas
technology possesses revolutionary potential to positively affect various domains of drug …

Recent advances of the biological and biomedical applications of CRISPR/Cas systems

Y Wang, C Huang, W Zhao - Molecular Biology Reports, 2022 - Springer
The clustered, regularly interspaced, short palindromic repeats (CRISPR)-associated
endonuclease (Cas) system, referred to as CRISPR/Cas system, has attracted significant …

An efficient isoelectric focusing of microcolumn array chip for screening of adult beta-thalassemia

G Zha, X Xiao, Y Tian, H Zhu, P Chen, Q Zhang, C Yu… - Clinica Chimica …, 2023 - Elsevier
Traditional capillary isoelectric focusing (cIEF), liquid chromatography (LC) and capillary
zone electrophoresis (CZE) still suffered from low resolution for hemoglobinopathy …

Impact of α-Globin Gene Expression and α-Globin Modifiers on the Phenotype of β-Thalassemia and Other Hemoglobinopathies: Implications for Patient Management

J Traeger-Synodinos, C Vrettou, C Sofocleous… - International Journal of …, 2024 - mdpi.com
In this short review, we presented and discussed studies on the expression of globin genes
in β-thalassemia, focusing on the impact of α-globin gene expression and α-globin modifiers …

CRISPR/Cas9, a promising approach for the treatment of β-thalassemia: A systematic review

A Khiabani, MH Kohansal, A Keshavarzi… - Molecular Genetics and …, 2023 - Springer
The CRISPR/Cas9 technique is easily programmable, fast, more powerful, and efficient at
generating a mutation compared to previous gene therapy methods. β-thalassemia is the …

Genetic manipulation strategies for β-thalassemia: a review

NA Zakaria, R Bahar, WZ Abdullah… - Frontiers in …, 2022 - frontiersin.org
Thalassemias are monogenic hematologic diseases that are classified as α-or β-
thalassemia according to its quantitative abnormalities of adult α-or β-globin chains. β …

Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment

I Rahimmanesh, M Boshtam, S Kouhpayeh… - Biology, 2022 - mdpi.com
Simple Summary β-thalassemia syndromes are clinically and genetically heterogeneous
blood disorders presented by β-chain deficiency in hemoglobin production. Despite …

Prime editing: A potential treatment option for β‐thalassemia

T Arif, A Farooq, FJ Ahmad, M Akhtar… - Cell Biology …, 2023 - Wiley Online Library
The potential to therapeutically alter the genome is one of the remarkable scientific
developments in recent years. Genome editing technologies have provided an opportunity …

[HTML][HTML] Investigating The Correction of IVS II-1 (G> A) Mutation in HBB Gene in TLS-12 Cell Line Using CRISPR/Cas9 System

N Servatian, S Abroun, SAS Fazeli… - Cell Journal …, 2023 - ncbi.nlm.nih.gov
Objective: Beta-thalassemia is a group of inherited hematologic. The most HBB gene variant
among Iranian beta-thalassemia patients is related to two mutations of IVSII-1 (G> A) and …