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Review
. 2020 Feb;22(2):e3150.
doi: 10.1002/jgm.3150. Epub 2020 Jan 20.

Gene transfer to skeletal muscle using hydrodynamic limb vein injection: current applications, hurdles and possible optimizations

Yann Thierry Le Guen  1 , Tony Le Gall  1 , Patrick Midoux  2 , Philippe Guégan  3 , Serge Braun  4 , Tristan Montier  1   5
Affiliations
Review

Gene transfer to skeletal muscle using hydrodynamic limb vein injection: current applications, hurdles and possible optimizations

Yann Thierry Le Guen et al. J Gene Med. 2020 Feb.

Abstract

Hydrodynamic limb vein injection is an in vivo locoregional gene delivery method. It consists of administrating a large volume of solution containing nucleic acid constructs in a limb with both blood inflow and outflow temporarily blocked using a tourniquet. The fast, high pressure delivery allows the musculature of the whole limb to be reached. The skeletal muscle is a tissue of choice for a variety of gene transfer applications, including gene therapy for Duchenne muscular dystrophy or other myopathies, as well as for the production of antibodies or other proteins with broad therapeutic effects. Hydrodynamic limb vein delivery has been evaluated with success in a large range of animal models. It has also proven to be safe and well-tolerated in muscular dystrophy patients, thus supporting its translation to the clinic. However, some possible limitations may occur at different steps of the delivery process. Here, we have highlighted the interests, bottlenecks and potential improvements that could further optimize non-viral gene transfer following hydrodynamic limb vein injection.

Keywords: gene transfer; hydrodynamic delivery; locoregional; non-viral gene delivery; optimizations; skeletal muscle.

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References

REFERENCES

    1. Braun S. Muscular gene transfer using nonviral vectors. Curr Gene Ther. 2008;8:391-405.
    1. Broderick KE, Humeau LM. Enhanced delivery of DNA or RNA vaccines by electroporation. Methods Mol Biol (Clifton, NJ). 2017;1499:193-200.
    1. Grzelkowska-Kowalczyk K. The importance of extracellular matrix in skeletal muscle development and function. In: Travascio F, ed. Composition and Function of the Extracellular Matrix in the Human Body. London, UK: IntechOpen Limited; 2016. https://doi.org/10.5772/62230
    1. Biga LM, Dawson S, Harwell A, et al. 10.2 Skeletal Muscle - Anatomy and Physiology. https://opentextbc.ca/anatomyandphysiology/chapter/10-2-skeletal-muscle/. Accessed July 24, 2019.
    1. Hagstrom JE, Hegge J, Zhang G, et al. A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Mol Ther J am Soc Gene Ther. 2004;10:386-398.

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