ReviewGene therapy2009
Progress and prospects: gene therapy for inherited immunodeficiencies.
Review in Gene therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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
23 citing papers in PubMed, 71 citations in OpenAlex.
- Variability in Genome Editing Outcomes: Challenges for Research Reproducibility and Clinical Safety.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Review
- Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency.Human gene therapy · 2017Article
- Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency.Human gene therapy · 2015Article
- Integration site and clonal expansion in human chronic retroviral infection and gene therapy.Viruses · 2014Review
- Combinatorial biomatrix/cell-based therapies for restoration of host tissue architecture and function.Advanced healthcare materials · 2013Review
- Review
- Crystallization and preliminary crystallographic analysis of an Escherichia coli-selected mutant of the nuclease domain of the metallonuclease colicin E7.Acta crystallographica. Section F, Structural biology and crystallization communications · 2013Article
- Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs.Gene therapy · 2013Article
- A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction.Annals of hematology · 2012Article
- Lentiviral vector induced insertional haploinsufficiency of Ebf1 causes murine leukemia.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Review
- Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression.Human gene therapy · 2012Article
- Genetically engineered immune privileged Sertoli cells: A new road to cell based gene therapy.Spermatogenesis · 2012Article
- Adipocytes as a vehicle for ex vivo gene therapy: Novel replacement therapy for diabetes and other metabolic diseases.Journal of diabetes investigation · 2011Review
- Artemis splice defects cause atypical SCID and can be restored in vitro by an antisense oligonucleotide.Genes and immunity · 2011Article
- Gene therapy for primary immunodeficiencies: looking ahead, toward gene correction.The Journal of allergy and clinical immunology · 2011Review
- Educational paper. The expanding clinical and immunological spectrum of severe combined immunodeficiency.European journal of pediatrics · 2011Review
- Therapeutic delivery of mRNA: the medium is the message.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases.Journal of immunological methods · 2011Review
- Gene therapy of chronic granulomatous disease: the engraftment dilemma.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
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 at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Haematopoietic stem cell transplantation (HSCT) is now widely used to treat primary immunodeficiencies (PID). For patients with specific disorders (severe combined immunodeficiency (SCID)-X1, adenosine deaminase deficiency (ADA)-SCID, X-chronic granulomatous disease (CGD) and Wiskott-Aldrich Syndrome (WAS)) who lack a suitable human leukocyte antigen (HLA)-matched donor, gene therapy has offered an important alternative treatment option. The success of gene therapy can be attributed, in part, to the selective advantage offered to gene-corrected cells, the avoidance of graft-versus-host disease and to the use of pre-conditioning in patients with chemotherapy to facilitate engraftment of corrected cells. Adverse events have been encountered and this has led to detailed characterization of retroviral vector integration profiles. A new generation of self-inactivating retroviral and lentiviral vectors have been designed to address these safety concerns, and are at an advanced stage of preparation for the next phase of clinical testing.
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.