What is recorded here?
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.
Source-linked catalogue note. A detailed explanatory guide has not yet been prepared for this entry.
Scientific scope
The 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.
Read the original work
- Frangoul et al., CRISPR–Cas9 editing for sickle-cell disease and β-thalassemia
- Crossref record for CRISPR hemoglobinopathy report
This English explanation is an editorial reading view. It does not replace the complete original work or certify its science.
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People and systems
Haydar 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
The named authors contributed to this scoped work; individual effort shares and later applications are not assigned by the byline.
Research locations and affiliations
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Documented countries / regions: United States, Italy, Germany, United Kingdom, Greece, Canada, France
Documented research locations or paper affiliations for this contribution. Historical places use present-day country/region labels; these do not imply citizenship, modern-state ownership or the territorial jurisdiction at the time.
English catalogue narrative
Frangoul and colleagues: CRISPR–Cas9 editing for sickle-cell disease and β-thalassemia
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.
Scientific scope
The 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.
Reading status
This 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.
People and systems
Haydar 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
The named authors contributed to this scoped work; individual effort shares and later applications are not assigned by the byline.