ReviewBlood2016
Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.
Review in Blood, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 73 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
73 citing papers in PubMed.
- Thalassaemia.Nature reviews. Disease primers · 2026Review
- Targeted attack: Harnessing myelin-specific plasmolipin for suppression of neuroblastoma and glioblastoma.Molecular therapy. Oncology · 2026Article
- Gene modification: Exploring the potential in treating kidney diseases.Pharmacological research · 2026Review
- Prognostic Value of MicroRNA Profiles in Septic Patients with Rare Hereditary Anemias: Insights from a Longitudinal Cohort Study.International journal of general medicine · 2026Article
- Ligase-mediated programmable genomic integration (L-PGI).Nature communications · 2025Article
- Combined haptoglobin and hemopexin therapy for the treatment of cardiopulmonary dysfunction in sickle cell disease.Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie · 2025Article
- In Silico Post-screening of Anti-polymerization Agents to Treat Sickle Cell Disease.bioRxiv : the preprint server for biology · 2025Article
- Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Innovations in Drug Discovery for Sickle Cell Disease Targeting Oxidative Stress and NRF2 Activation-A Short Review.International journal of molecular sciences · 2025Review
- Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches.Frontiers in pharmacology · 2025Review
- Revolutionary breakthrough: FDA approves CASGEVY, the first CRISPR/Cas9 gene therapy for sickle cell disease.Annals of medicine and surgery (2012) · 2024Review
- A new era of mutation rate analyses: Concepts and methods.Zoological research · 2024Review
- Pregnancy Outcomes Among Women With Sickle Cell Disease in Bahrain: A Case-Control Study.Cureus · 2024Article
- Article
- Inheritance of Hb S and G6PD deficiency in a familiar group.Archives of medical science : AMS · 2024Article
- Clinical-radiological-pathological correlation in pulmonary hypertension with unclear and/or multifactorial mechanisms.European respiratory review : an official journal of the European Respiratory Society · 2023Article
- Preferences for potential benefits and risks for gene therapy in the treatment of sickle cell disease.Blood advances · 2023Article
- Article
- Current challenges and new approaches to implementing optimal management of sickle cell disease in sub-Saharan Africa.Seminars in hematology · 2023Article
- Diverse Approaches to Gene Therapy of Sickle Cell Disease.Annual review of medicine · 2023Review
13 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent and severe monogenic disorder, SCD has been long considered a logical candidate for gene therapy. Significant progress has been made in moving toward this goal. These efforts have provided substantial insight into the natural regulation of the globin genes and illuminated challenges for genetic manipulation of the hematopoietic system. The initial γ-retroviral vectors, next-generation lentiviral vectors, and novel genome engineering and gene regulation approaches each share the goal of preventing erythrocyte sickling. After years of preclinical studies, several clinical trials for SCD gene therapies are now open. This review focuses on progress made toward achieving gene therapy, the current state of the field, consideration of factors that may determine clinical success, and prospects for future development.
Indexed as
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.