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

J Allergy Clin Immunol. 2017 May;139(5):1688-1692.e10. doi: 10.1016/j.jaci.2017.02.002. Epub 2017 Feb 17.
No abstract available

Publication types

  • Case Reports
  • Letter
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Africa
  • Alternative Splicing / genetics
  • Cell Proliferation / genetics
  • Consanguinity
  • Exome Sequencing
  • Genetic Therapy / methods*
  • Humans
  • Jurkat Cells
  • Male
  • Morpholinos / genetics*
  • Mutagenesis, Site-Directed
  • Mutation / genetics*
  • Pedigree
  • Severe Combined Immunodeficiency / genetics
  • Severe Combined Immunodeficiency / therapy*
  • Signal Transduction / genetics
  • T-Lymphocytes / physiology*
  • ZAP-70 Protein-Tyrosine Kinase / genetics*

Substances

  • Morpholinos
  • ZAP-70 Protein-Tyrosine Kinase
  • ZAP70 protein, human

Grants and funding