ReviewBlood2016
Customizing the genome as therapy for the β-hemoglobinopathies.
Review in Blood, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 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
29 citing papers in PubMed.
- Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative Medicine.Biomedicines · 2026Review
- Article
- Chemical engineering of CRISPR-Cas systems for therapeutic application.Nature reviews. Drug discovery · 2025Review
- Therapeutic gene correction of HBB frameshift CD41-42 (-TCTT) deletion in human hematopoietic stem cells.Advanced biotechnology · 2025Article
- Autologous gene therapy for hemoglobinopathies: From bench to patient's bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Review
- Reactivation of a developmentally silenced embryonic globin gene.Nature communications · 2021Article
- Genome editing to model and reverse a prevalent mutation associated with myeloproliferative neoplasms.PloS one · 2021Article
- Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies.Journal of biomedical research · 2020Article
- Gene Therapy of the Hemoglobinopathies.HemaSphere · 2020Review
- DNA Damage: From Threat to Treatment.Cells · 2020Review
- Directed evolution of adenine base editors with increased activity and therapeutic application.Nature biotechnology · 2020Article
- Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34Scientific reports · 2020Article
- The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements.Journal of clinical medicine · 2019Article
- The E3 ligase adaptor molecule SPOP regulates fetal hemoglobin levels in adult erythroid cells.Blood advances · 2019Article
- Omics Studies in Hemoglobinopathies.Molecular diagnosis & therapy · 2019Review
- TALEN-Mediated Gene Editing ofMolecular therapy. Methods & clinical development · 2019Article
- Orthogonal Cas9-Cas9 chimeras provide a versatile platform for genome editing.Nature communications · 2018Article
- Sickle Cell Anemia and Its Phenotypes.Annual review of genomics and human genetics · 2018Review
- β-Thalassemia intermedia: a comprehensive overview and novel approaches.International journal of hematology · 2018Review
- Sickle cell disease-Unanswered questions and future directions in therapy.Seminars in hematology · 2018Article
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
Abstract
Despite nearly complete understanding of the genetics of the β-hemoglobinopathies for several decades, definitive treatment options have lagged behind. Recent developments in technologies for facile manipulation of the genome (zinc finger nucleases, transcription activator-like effector nucleases, or clustered regularly interspaced short palindromic repeats-based nucleases) raise prospects for their clinical application. The use of genome-editing technologies in autologous CD34(+) hematopoietic stem and progenitor cells represents a promising therapeutic avenue for the β-globin disorders. Genetic correction strategies relying on the homology-directed repair pathway may repair genetic defects, whereas genetic disruption strategies relying on the nonhomologous end joining pathway may induce compensatory fetal hemoglobin expression. Harnessing the power of genome editing may usher in a second-generation form of gene therapy for the β-globin disorders.
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.