ReviewFrontiers in genome editing2020
A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies.
Review in Frontiers in genome editing, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.
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
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 18 citations in OpenAlex.
- Liver and renal biochemical profiles of people with sickle cell disease in Africa: a systematic review and meta-analysis of case-control studies.Systematic reviews · 2024Pooled it
- Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative Medicine.Biomedicines · 2026Review
- Expanding the Microbial Genomic Landscape and Biotechnological Applications of CRISPR-Cas Systems.Biology · 2026Review
- Haematological Profile of Patients with Sickle Cell Disease in the Acholi Sub-Region, Uganda.Journal of blood medicine · 2026Article
- Therapeutic gene correction of HBB frameshift CD41-42 (-TCTT) deletion in human hematopoietic stem cells.Advanced biotechnology · 2025Article
- "We Cure Sickle Cell Disease with Herbs": Perspectives of Herbal Medicine Practitioners Treating Sickle Cell Disease in the Acholi Sub-Region.Journal of multidisciplinary healthcare · 2025Article
- Engineering single-cycle MeV vector for CRISPR-Cas9 gene editing.Molecular therapy. Methods & clinical development · 2024Article
- Base editing of key residues in the BCL11A-XL-specific zinc finger domains derepresses fetal globin expression.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine.Genes & diseases · 2024Review
- In Silico Molecular Docking and Dynamics Simulation Analysis of Potential Histone Lysine Methyl Transferase Inhibitors for Managing β-Thalassemia.Molecules (Basel, Switzerland) · 2023Article
- Article
- The hemoglobinopathies, molecular disease mechanisms and diagnostics.International journal of laboratory hematology · 2022Review
- New Entity-Thalassemic Endocrine Disease: Major Beta-Thalassemia and Endocrine Involvement.Diagnostics (Basel, Switzerland) · 2022Review
- Development and clinical translation ofComputational and structural biotechnology journal · 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
7 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Throughout the past decades, the search for a treatment for severe hemoglobinopathies has gained increased interest within the scientific community. The discovery that ɤ-globin expression from intact
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.