Strimvelis

Strimvelis is the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency), a rare disorder caused by the absence of an essential protein called adenosine deaminase (ADA), which is required for the production of lymphocytes. ^[1]

4 relations: Adenosine deaminase deficiency, Gene therapy, List of Italian inventions, 2016 in science.

Adenosine deaminase deficiency

Adenosine deaminase deficiency (also called ADA deficiency or ADA-SCID) is an autosomal recessive metabolic disorder that causes immunodeficiency.

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Gene therapy

In the medicine field, gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease.

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List of Italian inventions

Italy has been the source of many significant inventions.

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2016 in science

A number of significant scientific events occurred in 2016.

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References

[1] https://en.wikipedia.org/wiki/Strimvelis

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