title: Morpholino-based correction of hypomorphic ZAP70 mutation in an adult with combined immunodeficiency.
authors.affiliation: Infectious Disease Susceptibility Program, McGill University Health Centre (MUHC) and Research Institute-MUHC (RI-MUHC), Montreal, Quebec, Canada.
authors.forename: Christina
authors.initials: C
authors.lastname: Gavino
authors.affiliation: Infectious Disease Susceptibility Program, McGill University Health Centre (MUHC) and Research Institute-MUHC (RI-MUHC), Montreal, Quebec, Canada.
authors.forename: Marija
authors.initials: M
authors.lastname: Landekic
authors.affiliation: Infectious Disease Susceptibility Program, McGill University Health Centre (MUHC) and Research Institute-MUHC (RI-MUHC), Montreal, Quebec, Canada.
authors.forename: Jibin
authors.initials: J
authors.lastname: Zeng
authors.affiliation: Laboratory of Molecular Oncology, Institut de recherches cliniques de Montréal (IRCM), Montreal, Quebec, Canada.
authors.forename: Ning
authors.initials: N
authors.lastname: Wu
authors.affiliation: Department of Pathology, MUHC, Montreal, Quebec, Canada.
authors.forename: Sungmi
authors.initials: S
authors.lastname: Jung
authors.affiliation: Laboratory of Molecular Oncology, Institut de recherches cliniques de Montréal (IRCM), Montreal, Quebec, Canada.
authors.forename: Ming-Chao
authors.initials: MC
authors.lastname: Zhong
authors.affiliation: Bioinformatics & Molecular Biology Core Facility, IRCM, Montreal, Quebec, Canada.
authors.forename: Alexis
authors.initials: A
authors.lastname: Cohen-Blanchet
authors.affiliation: Infectious Disease Susceptibility Program, McGill University Health Centre (MUHC) and Research Institute-MUHC (RI-MUHC), Montreal, Quebec, Canada.
authors.forename: Mélanie
authors.initials: M
authors.lastname: Langelier
authors.affiliation: Bioinformatics & Molecular Biology Core Facility, IRCM, Montreal, Quebec, Canada.
authors.forename: Odile
authors.initials: O
authors.lastname: Neyret
authors.affiliation: Department of Pediatrics, Division of Allergy & Clinical Immunology, MUHC, Montreal, Quebec, Canada.
authors.forename: Duncan
authors.initials: D
authors.lastname: Lejtenyi
authors.affiliation: Infectious Disease Susceptibility Program, McGill University Health Centre (MUHC) and Research Institute-MUHC (RI-MUHC), Montreal, Quebec, Canada.
authors.forename: Claudia
authors.initials: C
authors.lastname: Rochefort
authors.affiliation: Bioinformatics & Molecular Biology Core Facility, IRCM, Montreal, Quebec, Canada.
authors.forename: Judith
authors.initials: J
authors.lastname: Cotton-Montpetit
authors.affiliation: Department of Pediatrics, Division of Allergy & Clinical Immunology, MUHC, Montreal, Quebec, Canada.
authors.forename: Christine
authors.initials: C
authors.lastname: McCusker
authors.affiliation: Department of Pediatrics, Division of Allergy & Clinical Immunology, MUHC, Montreal, Quebec, Canada.
authors.forename: Bruce
authors.initials: B
authors.lastname: Mazer
authors.affiliation: Laboratory of Molecular Oncology, Institut de recherches cliniques de Montréal (IRCM), Montreal, Quebec, Canada.
authors.forename: André
authors.initials: A
authors.lastname: Veillette
authors.affiliation: Infectious Disease Susceptibility Program, McGill University Health Centre (MUHC) and Research Institute-MUHC (RI-MUHC), Montreal, Quebec, Canada; Laboratory of Molecular Oncology, Institut de recherches cliniques de Montréal (IRCM), Montreal, Quebec, Canada. Electronic address: donald.vinh@mcgill.ca.
authors.forename: Donald C
authors.initials: DC
authors.lastname: Vinh
mesh_terms: D000328:Adult; D000349:Africa; D017398:Alternative Splicing / Q000235:genetics; D049109:Cell Proliferation / Q000235:genetics; D003241:Consanguinity; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D019169:Jurkat Cells; D008297:Male; D060172:Morpholinos / Q000235:genetics*; D016297:Mutagenesis, Site-Directed; D009154:Mutation / Q000235:genetics*; D010375:Pedigree; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*; D015398:Signal Transduction / Q000235:genetics; D013601:T-Lymphocytes / Q000502:physiology*; D000073359:Exome Sequencing; D051746:ZAP-70 Protein-Tyrosine Kinase / Q000235:genetics*
chemical_list: D060172:Morpholinos; D051746:ZAP-70 Protein-Tyrosine Kinase; C497261:ZAP70 protein, human