ReviewGene therapy2010
Large animal models of hematopoietic stem cell gene therapy.
Review in Gene therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 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
38 citing papers in PubMed, 51 citations in OpenAlex.
- Spotlight on the impact of viral infections on Hematopoietic Stem Cells (HSCs) with a focus on COVID-19 effects.Cell communication and signaling : CCS · 2023Review
- Transplantation of human cells into Interleukin-2 receptor gamma gene knockout pigs under several conditions.Regenerative therapy · 2022Article
- CRISPR/Cas9 genome editing to create nonhuman primate models for studying stem cell therapies for HIV infection.Retrovirology · 2022Review
- Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.Pharmaceutics · 2022Review
- Genome editing in large animal models.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Transplantation of T-cell receptor α/β-depleted allogeneic bone marrow in nonhuman primates.Experimental hematology · 2021Article
- Developments and translational relevance for the canine haematopoietic cell transplantation preclinical model.Veterinary and comparative oncology · 2020Review
- Mouse models in hematopoietic stem cell gene therapy and genome editing.Biochemical pharmacology · 2020Review
- Modeling blood diseases with human induced pluripotent stem cells.Disease models & mechanisms · 2019Review
- MISTRG mice support engraftment and assessment of nonhuman primate hematopoietic stem and progenitor cells.Experimental hematology · 2019Article
- Animal Models for Preclinical Development of Allogeneic Hematopoietic Cell Transplantation.ILAR journal · 2018Article
- Placental Stem Cells from Domestic Animals: Translational Potential and Clinical Relevance.Cell transplantation · 2018Article
- Recombination activating gene-2Oncotarget · 2017Article
- Large-Scale Ex Vivo Generation of Human Red Blood Cells from Cord Blood CD34Stem cells translational medicine · 2017Article
- Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector.Molecular therapy. Methods & clinical development · 2017Article
- Safety and Efficacy of Megakaryocytes Induced from Hematopoietic Stem Cells in Murine and Nonhuman Primate Models.Stem cells translational medicine · 2017Article
- Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy.Nature communications · 2016Article
- GSK3β Inhibition Promotes Efficient Myeloid and Lymphoid Hematopoiesis from Non-human Primate-Induced Pluripotent Stem Cells.Stem cell reports · 2016Article
- Stem cell gene therapy for HIV: strategies to inhibit viral entry and replication.Current HIV/AIDS reports · 2015Review
- Natural Scaffolds for Renal Differentiation of Human Embryonic Stem Cells for Kidney Tissue Engineering.PloS one · 2015Article
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 at 2 institutions in 1 country.
Funding
Abstract
Large animal models have been instrumental in advancing hematopoietic stem cell (HSC) gene therapy. Here we review the advantages of large animal models, their contributions to the field of HSC gene therapy and recent progress in this field. Several properties of human HSCs including their purification, their cell-cycle characteristics, their response to cytokines and the proliferative demands placed on them after transplantation are more similar in large animal models than in mice. Progress in the development and use of retroviral vectors and ex vivo transduction protocols over the last decade has led to efficient gene transfer in both dogs and nonhuman primates. Importantly, the approaches developed in these models have translated well to the clinic. Large animals continue to be useful to evaluate the efficacy and safety of gene therapy, and dogs with hematopoietic diseases have now been cured by HSC gene therapy. Nonhuman primates allow evaluation of aspects of transplantation as well as disease-specific approaches such as AIDS (acquired immunodeficiency syndrome) gene therapy that can not be modeled well in the dog. Finally, large animal models have been used to evaluate the genotoxicity of viral vectors by comparing integration sites in hematopoietic repopulating cells and monitoring clonality after transplantation.
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.