exa-cel CRISPR sickle
Selected indexed studies
- Exagamglogene Autotemcel for Severe Sickle Cell Disease. (N Engl J Med, 2024) [PMID:38661449]
- Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia. (Med Sci Monit, 2024) [PMID:38425279]
- A roadmap for affordable genetic medicines. (Nature, 2024) [PMID:39019069]
_Worker-drafted node — pending editorial review._
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Sources
- Exagamglogene Autotemcel for Severe Sickle Cell Disease. (2024) pubmed
- Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia. (2024) pubmed
- A roadmap for affordable genetic medicines. (2024) pubmed
- CRISPR/Cas9 in the treatment of sickle cell disease (SCD) and its comparison with traditional treatment approaches: a review. (2024) pubmed
- CRISPR-based therapeutic genome editing for inherited blood disorders. (2025) pubmed
- CRISPR Therapeutics, Vertex Complete Rolling Biologics License Applications for Exa-Cel in Sickle Cell Disease, Beta Thalassemia. (2023) pubmed
- Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel. (2025) pubmed
- Exagamglogene Autotemcel: First Approval. (2024) pubmed
- An evaluation of exagamglogene autotemcel for the treatment of sickle cell disease and transfusion-dependent beta-thalassaemia. (2024) pubmed
- Infrequent Resolution of Vaso-Occlusive Crises in Routine Clinical Care Among Patients Mimicking the Exa-Cel Trial Population: A Cohort Study of Medicaid Enrollees. (2024) pubmed