ArticleMolecular therapy. Nucleic acids2019
ssODN-Mediated In-Frame Deletion with CRISPR/Cas9 Restores FVIII Function in Hemophilia A-Patient-Derived iPSCs and ECs.
Article in Molecular therapy. Nucleic acids, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed.
- Strategies and considerations for the generation of ssDNA-Based HDR templates for CRISPR-based genome editing.BMC genomics · 2026Review
- Exploring gene editing as a potential therapeutic strategy for hemophilia.Frontiers in bioengineering and biotechnology · 2026Review
- Exploiting urine-derived induced pluripotent stem cells for advancing precision medicine in cell therapy, disease modeling, and drug testing.Journal of biomedical science · 2024Review
- Identification of the Efficient Enhancer Elements in FVIII-Padua for Gene Therapy Study of Hemophilia A.International journal of molecular sciences · 2024Article
- Innovations in RNA therapy for hemophilia.Blood · 2023Review
- CRISPR/Cas9 Landscape: Current State and Future Perspectives.International journal of molecular sciences · 2023Review
- Ex vivo factor VIII-modified proliferating human hepatocytes therapy for haemophilia A.Cell proliferation · 2023Article
- Correction ofFrontiers in genetics · 2023Article
- A Novel Deletion Mutation of the F8 Gene for Hemophilia A.Diagnostics (Basel, Switzerland) · 2022Article
- Targeted-Deletion of a Tiny Sequence via Prime Editing to Restore SMN Expression.International journal of molecular sciences · 2022Article
- Targeted B-domain deletion restores F8 function in human endothelial cells and mice.Signal transduction and targeted therapy · 2022Article
- Review
- An update on precision genome editing by homology-directed repair in plants.Plant physiology · 2022Article
- Transcription factor POU3F2 regulates TRIM8 expression contributing to cellular functions implicated in schizophrenia.Molecular psychiatry · 2021Article
- CRISPR/Cas9-MediatedFrontiers in cell and developmental biology · 2021Article
- Restoration of FVIII Function and Phenotypic Rescue in Hemophilia A Mice by Transplantation of MSCs Derived FromFrontiers in cell and developmental biology · 2021Article
- Endothelial cells derived from patients' induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A.Stem cells translational medicine · 2020Article
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7 authors.
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Abstract
Given that the cDNA of F8 is too large to be packaged into adeno-associated virus (AAV) capsids, gene transfer of some versions of B-domain-deleted F8 (BDD-F8) for hemophilia A (HA) treatment has been attempted with promising results. Here, we describe an efficient gene correction via single-stranded-oligodeoxynucleotide (ssODN)-mediated in-frame deletion within the B domain of F8 with CRISPR/Cas9 in HA-patient-derived induced pluripotent stem cells (HA-iPSCs). The expression and activity of FVIII was restored in corrected HA-iPSC-derived induced endothelial progenitor cells (C-iEPCs) in vitro and in vivo. The bleeding phenotype was rescued in HA mice after C-iEPC infusion. Our results demonstrate an efficient approach for in situ gene correction via introduction of a tiny deletion using ssODN and CRISPR/Cas9 to reframe the F8 transcript and restore FVIII function in HA-iPSC-derived EPCs with potential clinical impact in HA gene therapy. For the first time, we demonstrated in vitro and in vivo the FVIII function that is encoded by the endogenous F8 gene with a partially deleted B domain. This work also suggests an applicable strategy for genetic correction of other gene frameshift mutations.
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