ReviewExpert opinion on biological therapy2011
Genotoxicity of retroviral hematopoietic stem cell gene therapy.
Review in Expert opinion on biological therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 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
28 citing papers in PubMed, 44 citations in OpenAlex.
- Safe CAR-T: shedding light on CAR-related T-cell malignancies.EMBO molecular medicine · 2025Article
- Efficacy and safety of a clinically relevant foamy vector design in human hematopoietic repopulating cells.The journal of gene medicine · 2018Article
- Ub-ISAP: a streamlined UNIX pipeline for mining unique viral vector integration sites from next generation sequencing data.BMC bioinformatics · 2017Article
- Engineering Next-Generation BET-Independent MLV Vectors for Safer Gene Therapy.Molecular therapy. Nucleic acids · 2017Article
- Evidence for the in vivo safety of insulated foamy viral vectors.Gene therapy · 2017Article
- Clinical Applications of Genome Editing to HIV Cure.AIDS patient care and STDs · 2016Article
- Retargeted Foamy Virus Vectors Integrate Less Frequently Near Proto-oncogenes.Scientific reports · 2016Article
- Review
- Review
- Insulated Foamy Viral Vectors.Human gene therapy · 2016Article
- Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy.World journal of stem cells · 2015Review
- A novel gammaretroviral shuttle vector insertional mutagenesis screen identifies SHARPIN as a breast cancer metastasis gene and prognostic biomarker.Oncotarget · 2015Article
- VISA--Vector Integration Site Analysis server: a web-based server to rapidly identify retroviral integration sites from next-generation sequencing.BMC bioinformatics · 2015Article
- Quantitative shearing linear amplification polymerase chain reaction: an improved method for quantifying lentiviral vector insertion sites in transplanted hematopoietic cell systems.Human gene therapy methods · 2015Article
- The therapeutic potential of genome editing for β-thalassemia.F1000Research · 2015Review
- A novel approach to identify driver genes involved in androgen-independent prostate cancer.Molecular cancer · 2014Article
- Molecular mechanisms of retroviral integration site selection.Nucleic acids research · 2014Article
- Identification of Hematopoietic Stem Cell Engraftment Genes in Gene Therapy Studies.Journal of stem cell research & therapy · 2013Article
- Development of a novel adenovirus-alphavirus hybrid vector with RNA replicon features for malignant hematopoietic cell transduction.Cancer gene therapy · 2013Article
- Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits.Journal of inherited metabolic disease · 2013Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
introductionRetroviral vectors have been developed for hematopoietic stem cell (HSC) gene therapy and have successfully cured X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), adrenoleukodystrophy, and Wiskott-Aldrich syndrome. However, in HSC gene therapy clinical trials, genotoxicity mediated by integrated vector proviruses has led to clonal expansion, and in some cases frank leukemia. Numerous studies have been performed to understand the molecular basis of vector-mediated genotoxicity with the aim of developing safer vectors and safer gene therapy protocols. These genotoxicity studies are critical to advancing HSC gene therapy. AREAS COVERED: This review provides an introduction to the mechanisms of retroviral vector genotoxicity. It also covers advances over the last 20 years in designing safer gene therapy vectors, and in integration site analysis in clinical trials and large animal models. Mechanisms of retroviral-mediated genotoxicity, and the risk factors that contribute to clonal expansion and leukemia in HSC gene therapy are introduced. EXPERT OPINION: Continued research on virus-host interactions and next-generation vectors should further improve the safety of future HSC gene therapy vectors and protocols.
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.