Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing f...
Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome
About this item
Full title
Author / Creator
Vavassori, Valentina , Mercuri, Elisabetta , Marcovecchio, Genni E , Castiello, Maria C , Schiroli, Giulia , Albano, Luisa , Margulies, Carrie , Buquicchio, Frank , Fontana, Elena , Beretta, Stefano , Merelli, Ivan , Cappelleri, Andrea , Rancoita, Paola MV , Lougaris, Vassilios , Plebani, Alessandro , Kanariou, Maria , Lankester, Arjan , Ferrua, Francesca , Scanziani, Eugenio , Cotta‐Ramusino, Cecilia , Villa, Anna , Naldini, Luigi and Genovese, Pietro
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
Precise correction of the
CD40LG
gene in T cells and hematopoietic stem/progenitor cells (HSPC) holds promise for treating X‐linked hyper‐IgM Syndrome (HIGM1), but its actual therapeutic potential remains elusive. Here, we developed a one‐size‐fits‐all editing strategy for effective T‐cell correction, selection, and depletion and investigated...
Alternative Titles
Full title
Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome
Authors, Artists and Contributors
Author / Creator
Mercuri, Elisabetta
Marcovecchio, Genni E
Castiello, Maria C
Schiroli, Giulia
Albano, Luisa
Margulies, Carrie
Buquicchio, Frank
Fontana, Elena
Beretta, Stefano
Merelli, Ivan
Cappelleri, Andrea
Rancoita, Paola MV
Lougaris, Vassilios
Plebani, Alessandro
Kanariou, Maria
Lankester, Arjan
Ferrua, Francesca
Scanziani, Eugenio
Cotta‐Ramusino, Cecilia
Villa, Anna
Naldini, Luigi
Genovese, Pietro
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_9aef4eba41804327af789ffa8b24647a
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_9aef4eba41804327af789ffa8b24647a
Other Identifiers
ISSN
1757-4676
E-ISSN
1757-4684
DOI
10.15252/emmm.202013545
