Morpholino-based correction of hypomorphic ZAP70 mutation in an adult with combined immunodeficiency

Publication date: Available online 17 February 2017
Source:Journal of Allergy and Clinical Immunology
Author(s): Christina Gavino, Marija Landekic, Jibin Zeng, Ning Wu, Sungmi Jung, Ming-Chao Zhong, Alexis Cohen-Blanchet, Mélanie Langelier, Odile Neyret, Duncan Lejtenyi, Claudia Rochefort, Judith Cotton-Montpetit, Christine McCusker, Bruce Mazer, André Veillette, Donald C. Vinh

Teaser

An adult with hypomorphic ZAP-70 deficiency due to de novo donor splice site mutation manifested autoimmunity, infections especially with DNA-based viruses and lymphoproliferation. Morpholino interference of mutant splice site increased expression of wild-type protein, significantly improving T cell responses.


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