ArticleHematology. American Society of Hematology. Education Program2021
Gene therapy for sickle cell disease: where we are now?
Article in Hematology. American Society of Hematology. Education Program, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
7 citing papers in PubMed.
- Gene Therapies for Sickle Cell Disease.The Journal of pharmacy technology : jPT : official publication of the Association of Pharmacy Technicians · 2024Review
- The value-based price of transformative gene therapy for sickle cell disease: a modeling analysis.Scientific reports · 2024Article
- Excellent outcome of stem cell transplantation for sickle cell disease.Annals of hematology · 2023Article
- A Framework for a Health Economic Evaluation Model for Patients with Sickle Cell Disease to Estimate the Value of New Treatments in the United States of America.PharmacoEconomics - open · 2023Article
- Long-term health outcomes following curative therapies for sickle cell disease.Hematology. American Society of Hematology. Education Program · 2022Article
- Surveillance for Sickle Cell Disease - Sickle Cell Data Collection Program, Two States, 2004-2018.Morbidity and mortality weekly report. Surveillance summaries (Washington, D.C. : 2002) · 2022Article
- Sickle Cell Disease in Children and Adolescents: A Review of the Historical, Clinical, and Public Health Perspective of Sub-Saharan Africa and Beyond.International journal of pediatrics · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The landscape of sickle cell disease (SCD) treatment continues to evolve rapidly, with new disease-modifying therapies in development and potentially curative options on the horizon. Until recently, allogeneic stem cell transplant has been the only proven cure for SCD. Gene therapy is rising to the forefront of the discussion as a potentially curative or highly disease- modifying option for abating the complications of the disease. Understanding the different types of gene therapy in use, the differences in their end points, and their potential risks and benefits will be key to optimizing the long-term use of this therapy.
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Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.