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

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 t...

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Bibliographic Details
Published in:The journal of gene medicine 2020-02, Vol.22 (2), p.e3150-n/a
Main Authors: Le Guen, Yann Thierry, Le Gall, Tony, Midoux, Patrick, Guégan, Philippe, Braun, Serge, Montier, Tristan
Format: Article
Language:English
Subjects:
Animal models
Animals
Duchenne's muscular dystrophy
Gene therapy
Gene transfer
Gene Transfer Techniques
Genetic Therapy - methods
Humans
hydrodynamic delivery
Hydrodynamics
Injection
Injections, Intravenous - methods
Life Sciences
locoregional
Muscle, Skeletal
Muscular dystrophy
Muscular Dystrophy, Duchenne - therapy
Musculoskeletal system
non‐viral gene delivery
optimizations
Skeletal muscle
Veins & arteries
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Summary: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.
ISSN:1099-498X
1521-2254
DOI:10.1002/jgm.3150