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DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID) (SCIDNET)

Date du début: 1 janv. 2016, Date de fin: 31 déc. 2019,

Severe combined immunodeficiency (SCID) is a devastating rare disorder of immune system development. Affected infants are born without functional immune systems and die within the first year of life unless effective treatment is given. Treatment options are limited to allogeneic haematopoietic stem cell transplantation and autologous stem cell gene therapy. Over the last 15 years, gene therapy for ...
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