Gene Therapy CDG

Our community is in a relentless pursuit of therapies!

Gene therapy is being considered for the following CDG types:

GNE-CDG (more information about this specific CDG type HERE, HERE, HERE and HERE)
PIGA-CDG (more information about this specific CDG type HERE and HERE)
SRD5A3-CDG (more information about this specific CDG type HERE, HERE HERE and HERE)

How can gene therapy help people living with GNE-CDG, PIGA-CDG and SRD5A3-CDG?

Gene therapy was studied in mouse models and cells of people living with GNE-CDG. Most of the gene therapy strategies use viruses to transport the normal DNA inside the cell, but this can have side effects. To minimize these effects, liposomes are being used to transport the DNA inside the cells of animal models and people living with GNE-CDG presenting more severe symptoms.

Gene therapy is also being studied in cells that have low levels of the PIGA gene with promising results. Further studies in an animal model will be performed once the model is available.

For SRD5A3-CDG, gene therapy will be tested in a mouse model with the same neurological symptoms as the people living with SRD5A3-CDG.

Publications in CDG are constantly growing. We have selected articles of special interest for you, see below. Not all are open access, thus if you wish to access the full information, please contact us at https://worldcdg.org/contact

CDG Therapies: From Bench to Bedside

Keep in mind, you are not alone. Though Congenital Disorders of Glycosylation (CDG) are categorized as “rare” there is an amazing community at your fingertips working day and night to improve the lives of many people living with CDG and their family members. We want you to know everything that is available about therapies in lay language, but we suggest you enter into it at your own pace and comfort level.

We are here to help
Should you need more details please do get in touch with our Team

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For further learning about CDG, move ahead to our community tailored sections across https://worldcdg.org/

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Authors

Vanessa Ferreira and Sandra Brasil (CDG & Allies FCT, NOVA University, World CDG Organization and Portuguese Association for CDG). Ines Santos, Tiago Martins, Madalena Raposo from Sci and Volunteer Program Nova School of Science and Technology 2021. Ana Sofia Rodrigues (content management, CDG & Allies FCT, NOVA University, World CDG Organization and Portuguese Association for CDG).

Disclaimer

The Site cannot and does not contain medical or health advice. The information is provided for general informational and educational purposes only and is not a substitute for professional advice.

Accordingly, before taking any actions based upon such information, we encourage you to consult with the appropriate professionals. We do not provide any kind of medical or health advice. The use or reliance of any information contained on this site is solely at your own risk.

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