Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo.
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ABSTRACT:
SUBMITTER: Asperti C
PROVIDER: S-EPMC10482894 | biostudies-literature | 2023 Sep
REPOSITORIES: biostudies-literature
Publications
Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated <i>in vitro</i> and <i>in vivo</i>.
Asperti Claudia C Canarutto Daniele D Porcellini Simona S Sanvito Francesca F Cecere Francesca F Vavassori Valentina V Ferrari Samuele S Rovelli Elisabetta E Albano Luisa L Jacob Aurelien A Sergi Sergi Lucia L Montaldo Elisa E Ferrua Francesca F González-Granado Luis Ignacio LI Lougaris Vassilios V Badolato Raffaele R Finocchi Andrea A Villa Anna A Radrizzani Marina M Naldini Luigi L
Molecular therapy. Methods & clinical development 20230823
Hyper-IgM1 is a rare X-linked combined immunodeficiency caused by mutations in the CD40 ligand (<i>CD40LG</i>) gene with a median survival of 25 years, potentially treatable with <i>in situ</i> CD4+ T cell gene editing with Cas9 and a one-size-fits-most corrective donor template. Here, starting from our research-grade editing protocol, we pursued the development of a good manufacturing practice (GMP)-compliant, scalable process that allows for correction, selection and expansion of edited cells, ...[more]
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