Recent advances in CRISPR/Cas9 delivery approaches for therapeutic gene editing of stem cells

M Lotfi, D Morshedi Rad, SS Mashhadi… - Stem cell reviews and …, 2023‏ - Springer
Rapid advancement in genome editing technologies has provided new promises for treating
neoplasia, cardiovascular, neurodegenerative, and monogenic disorders. Recently, the …

CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia

S Zeng, S Lei, C Qu, Y Wang, S Teng, P Huang - Human Genetics, 2023‏ - Springer
Abstract Beta-thalassemia (β-thalassemia) is an autosomal recessive disorder caused by
point mutations, insertions, and deletions in the HBB gene cluster, resulting in the …

Precision editing as a therapeutic approach for β-hemoglobinopathies

K Paschoudi, E Yannaki, N Psatha - International journal of molecular …, 2023‏ - mdpi.com
Beta-hemoglobinopathies are the most common genetic disorders worldwide, caused by a
wide spectrum of mutations in the β-globin locus, and associated with morbidity and early …

[HTML][HTML] Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients

LC Cosenza, J Gasparello, N Romanini, M Zurlo… - … Therapy Methods & …, 2021‏ - cell.com
Gene editing by the CRISPR-Cas9 nuclease system technology can be considered among
the most promising strategies to correct hereditary mutations in a variety of monogenic …

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 …

[HTML][HTML] Epigenetic Insights and Potential Modifiers as Therapeutic Targets in β–Thalassemia

NA Zakaria, MA Islam, WZ Abdullah, R Bahar… - Biomolecules, 2021‏ - mdpi.com
Thalassemia, an inherited quantitative globin disorder, consists of two types, α–and β–
thalassemia. β–thalassemia is a heterogeneous disease that can be asymptomatic, mild, or …

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 …

[HTML][HTML] CRISPR technology: A versatile tool to model, screen, and reverse drug resistance in cancer

S Shirani-Bidabadi, A Tabatabaee, N Tavazohi… - European Journal of …, 2023‏ - Elsevier
Background Drug resistance is a serious challenge in cancer treatment that can render
chemotherapy a failure. Understanding the mechanisms behind drug resistance and …

Genome editing approaches to β-hemoglobinopathies

M Brusson, A Miccio - Progress in molecular biology and translational …, 2021‏ - Elsevier
Abstract β-hemoglobinopathies are the most common monogenic disorders worldwide and
are caused by mutations in the β-globin locus altering the production of adult hemoglobin …

CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease

S Demirci, A Leonard, K Essawi, JF Tisdale - Molecular Therapy Methods & …, 2021‏ - cell.com
Genome editing is potentially a curative technique available to all individuals with β-
hemoglobinopathies, including sickle cell disease (SCD). Fetal hemoglobin (HbF) inhibits …