Generation of a Nonhuman Primate Model of Severe Combined Immunodeficiency Using Highly Efficient Genome Editing

Journal: Cell Stem Cell

Published: 2016-06-30

DOI: 10.1016/j.stem.2016.06.003

Affiliations: 11

Authors: 26

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Research Highlight

Marmoset models of a faulty immune system

© Floridapfe from S.Korea Kim in cherl/Moment Open/Getty

© Floridapfe from S.Korea Kim in cherl/Moment Open/Getty

Marmosets bred with an immune system disorder could pave the way for monkey models of specific genetic diseases.

Genetically modified rodents are useful for studying diseases but do not adequately mimic the human body. A team, including researchers from Keio University, used two genome editing techniques to breed marmosets carrying the immune deficiency disorder X-SCID, a rare disease that causes severe vulnerability to infections in young babies. Specially engineered enzymes were used to remove the X-SCID-associated gene, IL2-RG, from cultured cells. The team then injected the modified cells into marmoset embryos which were transferred to surrogate mothers. Nine marmosets, whose size makes them easy to work with, were born carrying the mutated gene, representing the first primate models of X-SCID.

New genome editing techniques like this could help researchers study a range of genetic disorders and investigate new therapies for X-SCID, the authors suggest.

Supported content

  1. Cell Stem Cell 9, 127-138 (2016). doi: 10.1016/j.stem.2016.06.003
Institutions Share
Central Institute for Experimental Animals, Japan 0.52
Keio University, Japan 0.13
Horizon Discovery - St. Louis, United States of America (USA) 0.08
Hiroshima University (HU), Japan 0.08
Juntendo University Nerima Hospital, Japan 0.04
Medical ProteoScope Co., Ltd. (MPS), Japan 0.04
Tokai University, Japan 0.04
Sagamihara National Hospital, Japan 0.04
Juntendo University, Japan 0.04