The DOI matches its Crossref record; 69 of the 70 references it cites were found in a registry or an index, and 1 are not checked yet.
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
Open accessAbstract
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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Cites
Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
Fu, Y., Sander, J. D., Reyon, D., Cascio, V. M., & Joung, J. K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2808 · Cited by 1,993
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Cavazzana-Calvo, M., Payen, E., Negre, O., Wang, G., Hehir, K., Fusil, F., Down, J., Denaro, M., Brady, T., Westerman, K., et al. (2010) · Nature · doi:10.1038/nature09328 · Cited by 1,276
Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific Repressor BCL11A
Sankaran, V. G., Menne, T. F., Xu, J., Akie, T. E., Lettre, G., Van Handel, B., Mikkola, H. K. A., Hirschhorn, J. N., Cantor, A. B., & Orkin, S. H. (2008) · Science · doi:10.1126/science.1165409 · Cited by 919
BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
Canver, M. C., Smith, E. C., Sher, F., Pinello, L., Sanjana, N. E., Shalem, O., Chen, D. D., Schupp, P. G., Vinjamur, D. S., Garcia, S. P., et al. (2015) · Nature · doi:10.1038/nature15521 · Cited by 886
Self-citationCRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
Dever, D. P., Bak, R. O., Reinisch, A., Camarena, J., Washington, G., Nicolas, C. E., Pavel-Dinu, M., Saxena, N., Wilkens, A. B., Mantri, S., et al. (2016) · Nature · doi:10.1038/nature20134 · Cited by 864
Self-citationQuantitative analysis of chromosome conformation capture assays (3C-qPCR)
Hagège, H., Klous, P., Braem, C., Splinter, E., Dekker, J., Cathala, G., de Laat, W., & Forné, T. (2007) · Nature Protocols · doi:10.1038/nprot.2007.243 · Cited by 720
Gene therapy in a patient with sickle cell disease
Ribeil, J.-A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., Caccavelli, L., Neven, B., Bourget, P., El Nemer, W., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1609677 · Cited by 696
Self-citationEx vivo generation of fully mature human red blood cells from hematopoietic stem cells
Giarratana, M., Kobari, L., Lapillonne, H., Chalmers, D., Kiger, L., Cynober, T., Marden, M. C., Wajcman, H., & Douay, L. (2004) · Nature Biotechnology · doi:10.1038/nbt1047 · Cited by 576
Targeted genome editing in human repopulating haematopoietic stem cells
Genovese, P., Schiroli, G., Escobar, G., Di Tomaso, T., Firrito, C., Calabria, A., Moi, D., Mazzieri, R., Bonini, C., Holmes, M. C., et al. (2014) · Nature · doi:10.1038/nature13420 · Cited by 555
Fetal hemoglobin in sickle cell anemia
Akinsheye, I., Alsultan, A., Solovieff, N., Ngo, D., Baldwin, C. T., Sebastiani, P., Chui, D. H. K., & Steinberg, M. H. (2011) · Blood · doi:10.1182/blood-2011-03-325258 · Cited by 553
Cited by
Sorted by citations
The promise and challenge of therapeutic genome editing
Doudna, J. A. (2020) · Nature · doi:10.1038/s41586-020-1978-5 · Cited by 1,126
Taher, A. T., Musallam, K. M., & Cappellini, M. D. (2021) · New England Journal of Medicine · doi:10.1056/nejmra2021838 · Cited by 507
CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations
Cullot, G., Boutin, J., Toutain, J., Prat, F., Pennamen, P., Rooryck, C., Teichmann, M., Rousseau, E., Lamrissi-Garcia, I., Guyonnet-Duperat, V., et al. (2019) · Nature Communications · doi:10.1038/s41467-019-09006-2 · Cited by 474
Farmakis, D. T., Porter, J. B., Taher, A. T., Cappellini, M. D., Angastiniotis, M., Eleftheriou, A., & Taskforce, F. T. 2. T. G. (2022) · HemaSphere · doi:10.1097/hs9.0000000000000732 · Cited by 346
Gene therapy using haematopoietic stem and progenitor cells
Ferrari, G., Thrasher, A. J., & Aiuti, A. (2020) · Nature Reviews Genetics · doi:10.1038/s41576-020-00298-5 · Cited by 263
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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