In vivo gene transfer of endo‐β‐galactosidase C removes αGal antigen on erythrocytes and endothelial cells of the organs

Miki, Yusuke; Maruyama, Shoichi; Liu, Da Ge; Kobayashi, Takaaki; Satô, Fumihiko; Shimizu, Hideaki; Kato, Shinichi; Sato, Waichi; Morita, Yoshiharu; Muramatsu, Takashi; Matsuo, Seiichi

doi:10.1111/j.1399-3089.2004.00163.x

Cited by 15 publications

(9 citation statements)

References 26 publications

(37 reference statements)

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“…For this purpose, CTLA4Ig cDNA was injected in vein, achieving high protein expression that allowed lengthening of graft survival time. Another approach involving the hydrodynamic procedure applied to organ xenotransplantation was described by Miki et al [ 110 ] and consisted of depleting the αGal protein from erythrocytes and kidney by injecting Igκ-EndoGalC (a protein with the capacity to remove αGal). The protein was expressed, with elimination of αGal from the vascular endothelium and digestion of 97% of the protein from erythrocytes for seven days.…”

Section: Translation Of the Hydrodynamic Method: From Mouse To Larmentioning

confidence: 99%

Translational Advances of Hydrofection by Hydrodynamic Injection

Sendra

Herrero

Aliño

2018

Genes

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Hydrodynamic gene delivery has proven to be a safe and efficient procedure for gene transfer, able to mediate, in murine model, therapeutic levels of proteins encoded by the transfected gene. In different disease models and targeting distinct organs, it has been demonstrated to revert the pathologic symptoms and signs. The therapeutic potential of hydrofection led different groups to work on the clinical translation of the procedure. In order to prevent the hemodynamic side effects derived from the rapid injection of a large volume, the conditions had to be moderated to make them compatible with its use in mid-size animal models such as rat, hamster and rabbit and large animals as dog, pig and primates. Despite the different approaches performed to adapt the conditions of gene delivery, the results obtained in any of these mid-size and large animals have been poorer than those obtained in murine model. Among these different strategies to reduce the volume employed, the most effective one has been to exclude the vasculature of the target organ and inject the solution directly. This procedure has permitted, by catheterization and surgical procedures in large animals, achieving protein expression levels in tissue close to those achieved in gold standard models. These promising results and the possibility of employing these strategies to transfer gene constructs able to edit genes, such as CRISPR, have renewed the clinical interest of this procedure of gene transfer. In order to translate the hydrodynamic gene delivery to human use, it is demanding the standardization of the procedure conditions and the molecular parameters of evaluation in order to be able to compare the results and establish a homogeneous manner of expressing the data obtained, as ‘classic’ drugs.

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Section: Translation Of the Hydrodynamic Method: From Mouse To Larmentioning

confidence: 99%

Translational Advances of Hydrofection by Hydrodynamic Injection

Sendra

Herrero

Aliño

2018

Genes

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“…The results are shown as number per 0.432 mm 2 . Four high-power fields (ϫ100) from each section were photographed, and areas positively stained for ␣-SMA or type III collagen in the submesothelial area of the peritoneum (above the muscle layer) were measured by computer-aided planimetry using Mac Scope (Mitani, Fukui, Japan) (22,28).…”

Section: Methodsmentioning

confidence: 99%

Tissue-type plasminogen activator deficiency attenuates peritoneal fibrosis in mice

Kurata¹,

Maruyama²,

Kato³

et al. 2009

American Journal of Physiology-Renal Physiology

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“…Chen and Woo demonstrated a complete cure of phenylketonuria (PKU) in PKU mice by hydrodynamic injection of plasmid containing phenylalanine hydroxylase gene (52). Additional examples include the use of hydrodynamic gene delivery to demonstrate therapeutic effect of the human growth hormone gene in hypophysectomized mice (53); arylsulfatase A and formylglycine-generating enzyme genes for metachromatic leukodystrophy (54); α-L-iduronidase gene for mucopolysaccharidosis I disease (55); short-chain acyl-CoA dehydrogenase gene for short chain acyl-CoA dehydrogenase deficiency (56); α-1 antitrypsin gene for treatment of alpha 1 deficiency (17, 19); insulin gene for diabetes (57); hepatocyte growth factor gene for diabetic nephropathy (58); uncoupling protein 1 gene (59) and leptin or ciliary neurotrophic factor genes 60) for obesity; interleukin (IL)-22-immunoglobulin (61), IL-13-immunoglobulin (62) and IL-1 receptor antagonist-immunoglobulin (63) fusion genes for myocarditis; IL-10 gene for glomerulonephritis (64); paraoxonase gene for reducing organophosphate toxicity (65); endo-beta-galactosidase C (66) and cytotoxic T-lymphocyte antigen 4-immunoglobulin (67) genes for prolonging the survival of xenotransplantation; eritropoietin gene for hypoxic-ischemia encephalopathy (68); nitric oxide synthase-2 gene for liver regeneration (69); and cytokine genes for cancer treatment (70–83). …”

Section: Hydrodynamic Delivery For Gene Drug Discoverymentioning

confidence: 99%

Hydrodynamic Gene Delivery and Its Applications in Pharmaceutical Research

2010

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Hydrodynamic delivery has emerged as the simplest and most effective method for intracellular delivery of membrane impermeable substances in rodents. The system employs a physical force generated by a rapid injection of large volume of solution into a blood vessel to enhance the permeability of endothelium and the plasma membrane of the parenchyma cells to allow delivery of substance into cells. The procedure was initially established for gene delivery in mice and its applications have been extended to the delivery of proteins, oligo nucleotides, genomic DNA and RNA sequences, and small molecules. The focus of this review is on applications of hydrodynamic delivery in pharmaceutical research. Examples are provided to highlight the use of hydrodynamic delivery for study of transcriptional regulation of CYP enzymes, for establishment of animal model for viral infections, and for gene drug discovery and gene function analysis.

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In vivo gene transfer of endo‐β‐galactosidase C removes αGal antigen on erythrocytes and endothelial cells of the organs

Cited by 15 publications

References 26 publications

Translational Advances of Hydrofection by Hydrodynamic Injection

Translational Advances of Hydrofection by Hydrodynamic Injection

Tissue-type plasminogen activator deficiency attenuates peritoneal fibrosis in mice

Hydrodynamic Gene Delivery and Its Applications in Pharmaceutical Research

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