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Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
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Key Points: CRISPR/Cas9-mediated disruption of the β-globin locus architecture reactivates fetal γ-globin expression in adult erythroblasts. Fetal γ-globin reactivation and sickle β-globin downregulation leads to the amelioration of the SCD cell phenotype.
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“The first involves restoration of the normal β -globin gene sequence by homology-directed repair 35 .”
From The promise and challenge of therapeutic genome editing (2020) · cites this paper as [35]
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Metadata
- Authors
- Antoniani, C., Meneghini, V., Lattanzi, A., Felix, T., Romano, O., Magrin, E., Weber, L., Pavani, G., El Hoss, S., Kurita, R., Nakamura, Y., Cradick, T. J., Lundberg, A. S., Porteus, M., Amendola, M., El Nemer, W., Cavazzana, M., Mavilio, F., & Miccio, A.
- Affiliations
- Inserm (FR)
- Laboratory of Chromatin and Gene Regulation during Development, Imagine Institute, INSERM Unité Mixte de Recherche (UMR)1163, Paris, France;
- University of Modena and Reggio Emilia (IT)
- Hôpital Necker-Enfants Malades (FR)
- Sorbonne Paris Cité University, Paris Diderot University, INSERM, Institut National de la Transfusion Sanguine, Unité Biologie Intégrée du Globule Rouge, Laboratoire d’Excellence GR-Ex, Paris, France;
- RIKEN (JP)
- CRISPR Therapeutics, Cambridge, MA;
- Stanford Medicine (US)
- Corresponding author
- Antoniani, Chiara
- Meneghini, Vasco
- ORCID
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2018-04-26
- Volume
- 131
- Issue
- 17
- Pages
- 1960-1973
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2793205487
- Cited by
- 194
- References
- 69
- Citations by year
- 2026: 10 · 2025: 8 · 2024: 18 · 2023: 24 · 2022: 22 · 2021: 39 · 2020: 24 · 2019: 26 · 2018: 23
- FWCI
- 15.84
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- other-oa
- Locations
- Blood (publishedVersion, other-oa)
- PubMed (publishedVersion)
- HAL (submittedVersion, other-oa)
- IRIS UNIMORE (University of Modena and Reggio Emilia) (submittedVersion, cc-by)
- HAL (submittedVersion)
- Archivio Istituzionale della Ricerca - Università Vita-Salute San Raffaele (submittedVersion)
- Padua Research Archive (University of Padova) (submittedVersion)
- Funders
- Agence Nationale de la Recherche (ANR-10-IAHU- 01, ANR-16-CE18-0004, ANR-11-LABX-0051, ANR-10-IAHU-0001, ANR-16, 10-IAHU-01, ANR-10)
- Topics
- Health Sciences › Medicine › Genetics (clinical) › Hemoglobinopathies and Related Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- fetal hemoglobin reactivation, CRISPR/Cas9 genome editing, beta-globin locus, sickle cell disease, beta-thalassemia, hereditary persistence of fetal hemoglobin, γ-globin expression, delta-globin gene, hematopoietic stem and progenitor cells, epigenetic modifications, chromatin interactions, β-hemoglobinopathies
- MeSH terms
- Gene Editing, Anemia, Sickle Cell, Cell Line, Fetal Hemoglobin, Hematopoietic Stem Cells, Humans, beta-Globins, Genetic Loci, CRISPR-Cas Systems
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