Peer-reviewed veterinary case report
Dog with cyclic neutropenia treated by gene therapy for 18 months
By Yanay, Ofer et al.·Published in Human gene therapy·2006·Department of Pediatrics, United States·View original on PubMed →
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Original publication title: An adult dog with cyclic neutropenia treated by lentivirus- mediated delivery of granulocyte colony-stimulating factor.
- Species:
- dog
Plain-English summary
A gray collie dog with cyclic neutropenia, a condition that causes low white blood cell counts, was treated with a special therapy that delivered a growth factor to boost his immune system. After receiving the treatment, the dog's neutrophil levels increased significantly, and he remained healthy for nearly 18 months without needing daily injections. He gained weight, showed no signs of infection, and could live normally without being isolated from other animals. This innovative treatment could help other dogs with similar blood disorders in the future.
People also search for: gray collie cyclic neutropenia treatment · dog low white blood cell count · immune system booster for dogs
Abstract
Cyclic neutropenia occurs in humans and gray collie dogs, is characterized by recurrent neutropenia, and is treated by daily injections of recombinant granulocyte colony-stimulating factor (G-CSF). After showing that canine recombinant G-CSF increased neutrophil counts in an affected dog, we administered intramuscularly 2 x 10(9) infectious units (IU) of a lentiviral vector encoding canine G-CSF cDNA. Elevated, therapeutic neutrophil production was obtained for nearly 18 months. Lentiviral vector treatment provided a mean neutrophil count of 29,230 +/- 12,930 cells/microl, which was significantly increased over both the pretreatment value (5,240 +/- 4,800 cells/microl; p < 0.0001) and the neutrophil count during G-CSF administration (17,820 +/- 11,100 cells/microl; p < 0.0001). By systemic infusion of recombinant G-CSF to normal dogs we estimated that 2 x 10(9) IU of lentivirus delivered 3.5 microg of G-CSF per kilogram per day. After lentiviral vector treatment the gray collie gained weight, showed no clinical signs of infection and fever, and no longer needed housing in a pathogen-free environment. Genomic DNA harvested from muscle at the injection sites was positive for provirus, whereas gonad, lung, spleen, heart, liver, kidney, and noninjected muscle samples were negative. These studies show that an adult animal is responsive long-term to lentivirus-mediated G-CSF delivery, suggesting this approach may be applied for treatment of adult patients with cyclic and other neutropenias.
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Search related cases →Original publication on PubMed: https://pubmed.ncbi.nlm.nih.gov/16610934/