title: Stem cell transplantation for tetratricopeptide repeat domain 7A deficiency: long-term follow-up.
authors.affiliation: Genetics and Genomic Medicine, Institute of Child Health, University College London, London, United Kingdom;
authors.forename: Jochen
authors.initials: J
authors.lastname: Kammermeier
authors.affiliation: Department of Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Giovanna
authors.initials: G
authors.lastname: Lucchini
authors.affiliation: Division of Hematology-Oncology, Boston Children's Hospital, Boston, MA;
authors.forename: Sung-Yun
authors.initials: SY
authors.lastname: Pai
authors.affiliation: Department of Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Austen
authors.initials: A
authors.lastname: Worth
authors.affiliation: Department of Histopathology, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Dyanne
authors.initials: D
authors.lastname: Rampling
authors.affiliation: Department of Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Persis
authors.initials: P
authors.lastname: Amrolia
authors.affiliation: Department of Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Juliana
authors.initials: J
authors.lastname: Silva
authors.affiliation: Department of Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Robert
authors.initials: R
authors.lastname: Chiesa
authors.affiliation: Department of Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Kanchan
authors.initials: K
authors.lastname: Rao
authors.affiliation: Department of Paediatric Gastroenterology, Addenbrooke's Hospital, Cambridge, United Kingdom;
authors.forename: Gabriele
authors.initials: G
authors.lastname: Noble-Jamieson
authors.affiliation: Department of Paediatric Gastroenterology, Addenbrooke's Hospital, Cambridge, United Kingdom;
authors.forename: Marco
authors.initials: M
authors.lastname: Gasparetto
authors.affiliation: North East Thames Regional Genetics Laboratory, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Drew
authors.initials: D
authors.lastname: Ellershaw
authors.affiliation: Transitional Gastroenterology Unit, Nuffield Department of Medicine and Department of Paediatrics, University of Oxford, Oxford, United Kingdom;
authors.forename: Holm
authors.initials: H
authors.lastname: Uhlig
authors.affiliation: Department of Histopathology, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Neil
authors.initials: N
authors.lastname: Sebire
authors.affiliation: Department of Gastroenterology, Great Ormond Street Hospital, London, United Kingdom; and.
authors.forename: Mamoun
authors.initials: M
authors.lastname: Elawad
authors.affiliation: Division of Immunology, Boston Children's Hospital, Boston, MA.
authors.forename: Luigi
authors.initials: L
authors.lastname: Notarangelo
authors.affiliation: Department of Gastroenterology, Great Ormond Street Hospital, London, United Kingdom; and.
authors.forename: Neil
authors.initials: N
authors.lastname: Shah
authors.affiliation: Department of Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital, London, United Kingdom;
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
mesh_terms: D005500:Follow-Up Studies; D020022:Genetic Predisposition to Disease / Q000235:genetics*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D015212:Inflammatory Bowel Diseases / Q000235:genetics / Q000628:therapy*; D009154:Mutation; D011506:Proteins / Q000235:genetics*; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D011506:Proteins; C583818:TTC7A protein, human