ArticleMolecular therapy. Nucleic acids2024
Context base editing for splice correction of IVSI-110 β-thalassemia.
Article in Molecular therapy. Nucleic acids, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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Who cites it
9 citing papers in PubMed.
- Therapeutic base editing alleviates restrictive cardiomyopathy.Cell reports. Medicine · 2026Article
- From Bench to Bedside: Ethical and Clinical Best Practices for Genome Editing Applications.International journal of molecular sciences · 2026Review
- Advances in Gene Therapy for Inherited Haemoglobinopathies.Hematology reports · 2025Review
- CRISPR/Cas-edited iPSCs and mesenchymal stem cells: a concise review of their potential in thalassemia therapy.Frontiers in cell and developmental biology · 2025Review
- Proceedings of the second annual meeting of GenE-HumDi (COST Action 21113).Frontiers in genome editing · 2025Article
- Editing of homologous globin genes by nickase-deficient base editor mitigates large intergenic deletions in HSPCs.Molecular therapy. Nucleic acids · 2024Article
- Gene Editing of the Endogenous Cryptic 3' Splice Site Corrects the RNA Splicing Defect in the βHuman gene therapy · 2024Article
- Precision genome editing offers hope for treatment of β-thalassemia and other genetic disorders.Molecular therapy. Nucleic acids · 2024Article
- Therapeutic Gene Editing for Hemoglobinopathies.Mediterranean journal of hematology and infectious diseases · 2024Review
Corrections and comments
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
β-Thalassemia is brought about by defective β-globin (HBB [hemoglobin subunit β]) formation and, in severe cases, requires regular blood transfusion and iron chelation for survival. Genome editing of hematopoietic stem cells allows correction of underlying mutations as curative therapy. As potentially safer alternatives to double-strand-break-based editors, base editors (BEs) catalyze base transitions for precision editing of DNA target sites, prompting us to reclone and evaluate two recently published adenine BEs (ABEs; SpRY and SpG) with relaxed protospacer adjacent motif requirements for their ability to correct the common
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Registered trials
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