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MATERIALS AND METHODS FOR TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY (SCID) OR OMENN SYNDROME
MATERIALS AND METHODS FOR TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY (SCID) OR OMENN SYNDROME
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机译：严重合并免疫缺陷病（OMED）或OMENN综合征的治疗材料和方法
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摘要
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摘要
The present application provides materials and methods for treating a patient with severe combined immunodeficiency (SCID) or Omenn Syndrome, both ex vive and in vive. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the Recombination Activating Gene 1 (RAG1) gene in a cell by genome editing.
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