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      "statement": "# Frangoul and colleagues: CRISPR–Cas9 editing for sickle-cell disease and β-thalassemia\n\nReported early clinical outcomes after ex vivo CRISPR–Cas9 disruption of a BCL11A erythroid enhancer in autologous hematopoietic stem and progenitor cells for one patient with transfusion-dependent β-thalassemia and one with sickle-cell disease.\n\n## Scientific scope\n\nThe record is the exact 2021 NEJM report of two treated patients and its stated follow-up. The CRISPR mechanism paper, later trial cohorts, regulatory approvals, manufacturing and claims of universal cure remain separate.\n\n## Reading status\n\nThis is a source-linked WRS catalogue note. A full explanatory guide and independent scientific reconstruction have not been completed for this entry. Read the identified original work for its complete narrative and arguments.\n\n## People and systems\n\nHaydar Frangoul; David Altshuler; M. Domenica Cappellini; Yi-Shan Chen; Jennifer Domm; Brenda K. Eustace; Juergen Foell; Josu de la Fuente; Stephan Grupp; Rupert Handgretinger; Tony W. Ho; Antonis Kattamis; Andrew Kernytsky; Julie Lekstrom-Himes; Amanda M. Li; Franco Locatelli; Markus Y. Mapara; Mariane de Montalembert; Damiano Rondelli; Akshay Sharma; Sujit Sheth; Sandeep Soni; Martin H. Steinberg; Donna Wall; Angela Yen; Selim Corbacioglu\n\nThe named authors contributed to this scoped work; individual effort shares and later applications are not assigned by the byline.\n\n\n\n## Original sources\n\n- [Frangoul et al., CRISPR–Cas9 editing for sickle-cell disease and β-thalassemia](https://www.nejm.org/doi/abs/10.1056/NEJMoa2031054)\n- [Crossref record for CRISPR hemoglobinopathy report](https://api.crossref.org/works/10.1056%2FNEJMoa2031054)\n",
      "summary": "Reported early clinical outcomes after ex vivo CRISPR–Cas9 disruption of a BCL11A erythroid enhancer in autologous hematopoietic stem and progenitor cells for one patient with transfusion-dependent β-thalassemia and one with sickle-cell disease.",
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    "title": "Frangoul and colleagues: CRISPR–Cas9 editing for sickle-cell disease and β-thalassemia",
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