Viral gene therapy could treat incurable disease

EU-funded researchers have conducted the first successful trials of an innovative gene therapy to treat a rare and debilitating metabolic disorder, using a small and innocuous virus as a carrier for genetic information to correct the functioning of liver and muscle cells.

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Countries
Countries
  Algeria
  Argentina
  Australia
  Austria
  Bangladesh
  Belarus
  Belgium
  Benin
  Bolivia
  Bosnia and Herzegovina
  Brazil
  Bulgaria
  Burkina Faso
  Cambodia
  Cameroon
  Canada
  Cape Verde
  Chile
  China
  Colombia
  Costa Rica
  Croatia
  Cyprus
  Czechia
  Denmark
  Ecuador
  Egypt
  Estonia
  Ethiopia
  Faroe Islands
  Finland
  France
  French Polynesia
  Georgia


  Infocentre

Published: 3 April 2019  
Related theme(s) and subtheme(s)
Health & life sciencesGenetic engineering  |  Medical research  |  Molecular biology  |  Rare & orphan diseases
Human resources & mobilityMarie Curie Actions
Research policyHorizon 2020
Countries involved in the project described in the article
France
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Viral gene therapy could treat incurable disease

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© vchalup #141880562, 2019 source: stock.adobe.com

The GLYCODIS3 project laid the foundations for a potential cure for glycogen storage disease type III (GSD III). This currently incurable genetic disorder causes generalised muscle deterioration. Triggered by excess accumulation of energy-storing glycogen molecules, the symptoms of GSD III can only currently be alleviated by patients following a strict diet of frequent meals high in carbohydrates to keep their blood sugar levels elevated, slowing but not stopping disease progression.

In new proof-of-concept laboratory trials, GLYCODIS3 researcher Giuseppe Ronzitti, head of the metabolic diseases laboratory at Genethon in France, demonstrated it is possible to modify the genes responsible for causing GSD III. The modifications enable glycogen to be cleared from muscle cells, thereby restoring muscle strength and helping to keep blood sugar in circulation.

The gene expression changes were achieved by encoding an adeno-associated virus, a small virus infectious to humans that is not known to have harmful effects, as a carrier for the gene therapy. Because the virus can only carry genetic information equivalent to around 5 000 base pairs of DNA, the researchers successfully engineered a novel dual-vector system that would carry different sets of genetic instructions separately and recombine once inside the target cells.

Adeno-associated viruses are currently the vectors of choice for gene therapy treatments and have been used in promising human trials – of liver gene transfer therapies to treat haemophilia, and eye gene transfer to treat congenital blindness. The work of Ronzitti and his team, which has led to five patents and numerous scientific publications, has further expanded the potential, not only for treating GSD III but several other neuromuscular disorders.

GLYCODIS3, which was supported by the EU’s Marie Skłodowska-Curie Individual Fellowships programme, has led to a number of follow-up projects that aim to continue the development of adeno-associated virus gene therapy for GSD III toward clinical use.

Project details

  • Project acronym: GLYCODIS3
  • Participants: France (Coordinator)
  • Project N°: 658712
  • Total costs: € 173 076
  • EU contribution: € 173 076
  • Duration: January 2016 to December 2017

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