ArticleBlood2006
Correction of the disease phenotype in canine leukocyte adhesion deficiency using ex vivo hematopoietic stem cell gene therapy.
Article in Blood, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01212055 (Apheresis and CD34+ Selection of Mobilized Peripheral Blood CD34+ Cells From Patients With DOCK8 Deficiency, LAD-1, and GATA2 Deficiency), which is not on this map. Cited by 26 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.
Apheresis and CD34+ Selection of Mobilized Peripheral Blood CD34+ Cells From Patients With DOCK8 Deficiency, LAD-1, and GATA2 Deficiency
Who cites it
26 citing papers in PubMed, 43 citations in OpenAlex.
- Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.Nature nanotechnology · 2024Article
- Evolution of haematopoietic cell transplantation for canine blood disorders and a platform for solid organ transplantation.Veterinary medicine and science · 2021Review
- Gene Therapies for Primary Immune Deficiencies.Frontiers in immunology · 2021Review
- Foamy Virus Vector Carries a Strong Insulator in Its Long Terminal Repeat Which Reduces Its Genotoxic Potential.Journal of virology · 2018Article
- Feline leukocyte adhesion (CD18) deficiency caused by a deletion in the integrin βVeterinary clinical pathology · 2017Article
- Advances of gene therapy for primary immunodeficiencies.F1000Research · 2016Review
- Domestic animals as models for biomedical research.Upsala journal of medical sciences · 2016Review
- Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases.International journal of hematology · 2014Review
- Long-term follow-up of foamy viral vector-mediated gene therapy for canine leukocyte adhesion deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
- Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models.Seminars in hematology · 2013Review
- Large animal models for foamy virus vector gene therapy.Viruses · 2012Review
- Counting stem cells: methodological constraints.Nature methods · 2012Article
- Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity.Frontiers of medicine · 2011Review
- Gene therapy for canine leukocyte adhesion deficiency with lentiviral vectors using the murine stem cell virus and human phosphoglycerate kinase promoters.Human gene therapy · 2011Article
- Gene therapy of canine leukocyte adhesion deficiency using lentiviral vectors with human CD11b and CD18 promoters driving canine CD18 expression.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Large animal models of hematopoietic stem cell gene therapy.Gene therapy · 2010Review
- Lentiviral vectors incorporating a human elongation factor 1alpha promoter for the treatment of canine leukocyte adhesion deficiency.Gene therapy · 2010Article
- Development and use of DNA archives at veterinary teaching hospitals to investigate the genetic basis of disease in dogs.Journal of the American Veterinary Medical Association · 2009Review
- Potential large animal models for gene therapy of human genetic diseases of immune and blood cell systems.ILAR journal · 2009Article
- The usefulness of a perfect parasite.Gene therapy · 2008Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 3 institutions in 1 country.
Funding
Abstract
Canine leukocyte adhesion deficiency (CLAD) represents the canine counter-part of the human disease leukocyte adhesion deficiency (LAD). Defects in the leukocyte integrin CD18 adhesion molecule in both CLAD and LAD lead to recurrent, life-threatening bacterial infections. We evaluated ex vivo retroviral-mediated gene therapy in CLAD using 2 nonmyeloablative conditioning regimens--200 cGy total body irradiation (TBI) or 10 mg/kg busulfan--with or without posttransplantation immunosuppression. In 6 of 11 treated CLAD dogs, therapeutic levels of CD18(+) leukocytes were achieved. Conditioning with either TBI or busulfan allowed long-term engraftment, and immunosuppression was not required for efficacy. The percentage of CD18(+) leukocytes in the peripheral blood progressively increased over 6 to 8 months after infusion to levels ranging from 1.26% to 8.37% at 1-year follow-up in the 6 dogs. These levels resulted in reversal or moderation of the severe CLAD phenotype. Linear amplification-mediated polymerase chain reaction assays indicated polyclonality of insertion sites. These results describe ex vivo hematopoietic stem cell gene transfer in a disease-specific, large animal model using 2 clinically applicable conditioning regimens, and they provide support for the use of nonmyeloablative conditioning regimens in preclinical protocols of retroviral-mediated gene transfer for nonmalignant hematopoietic diseases such as LAD.
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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.