Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X...
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency
About this item
Full title
Author / Creator
De Ravin, Suk See , Liu, Siyuan , Sweeney, Colin L. , Brault, Julie , Whiting-Theobald, Narda , Ma, Michelle , Liu, Taylor , Choi, Uimook , Lee, Janet , O’Brien, Sandra Anaya , Quackenbush, Priscilla , Estwick, Tyra , Karra, Anita , Docking, Ethan , Kwatemaa, Nana , Guo, Shuang , Su, Ling , Sun, Zhonghe , Zhou, Sheng , Puck, Jennifer , Cowan, Morton J. , Notarangelo, Luigi D. , Kang, Elizabeth , Malech, Harry L. and Wu, Xiaolin
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
Formats
Publication information
Publisher
London: Nature Publishing Group UK
Subjects
More information
Scope and Contents
Contents
X-linked Severe Combined Immunodeficiency (SCID-X1) due to
IL2RG
mutations is potentially fatal in infancy where ‘emergency’ life-saving stem cell transplant may only achieve incomplete immune reconstitution following transplant. Salvage therapy SCID-X1 patients over 2 years old (NCT01306019) is a non-randomized, open-label, phase I/II clinic...
Alternative Titles
Full title
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency
Authors, Artists and Contributors
Author / Creator
Liu, Siyuan
Sweeney, Colin L.
Brault, Julie
Whiting-Theobald, Narda
Ma, Michelle
Liu, Taylor
Choi, Uimook
Lee, Janet
O’Brien, Sandra Anaya
Quackenbush, Priscilla
Estwick, Tyra
Karra, Anita
Docking, Ethan
Kwatemaa, Nana
Guo, Shuang
Su, Ling
Sun, Zhonghe
Zhou, Sheng
Puck, Jennifer
Cowan, Morton J.
Notarangelo, Luigi D.
Kang, Elizabeth
Malech, Harry L.
Wu, Xiaolin
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_1af0140e7668454fa4433677e4f0df76
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_1af0140e7668454fa4433677e4f0df76
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-022-31344-x
