The I-CreI meganuclease and its engineered derivatives: applications from cell modification to gene therapy

Autor: Sylvain Arnould, C. Delenda, Julianne Smith, Frédéric Pâques, G.H. Silva, Sylvestre Grizot, C. Desseaux

Publikováno v: Protein engineering, designselection : PEDS. 24(1-2)

Meganucleases (MNs) are highly specific enzymes that can induce homologous recombination in different types of cells, including mammalian cells. Consequently, these enzymes are used as scaffolds for the development of custom gene-targeting tools for

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0fb77653d67ca6d49a3058a02497a742
https://pubmed.ncbi.nlm.nih.gov/21047873

A lentiviral vector encoding the human Wiskott–Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice

Autor: Bucher-Laurent S, Anne Galy, Olivier Danos, Seye K, David-Alexandre Gross, William Vainchenker, Paule Opolon, Charrier S, C Delenda, Daniel Stockholm, Taveau M

Publikováno v: Gene Therapy
Gene Therapy, 2005, 12 (7), pp.597-606. ⟨10.1038/sj.gt.3302440⟩
Gene Therapy, Nature Publishing Group, 2005, 12(7), pp.597-606

International audience; Wiskott–Aldrich syndrome (WAS) is an immune deficiency with thrombopenia resulting from mutations in the WASP gene. This gene normally encodes the Wiskott–Aldrich syndrome protein (WASP), a major cytoskeletal regulator exp

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c03ef6d5af133018dc8d5738b9596673
https://hal.science/hal-03765538

Biosafety Issues in Lentivector Production

Autor: O. Danos, M. Audit, C. Delenda

Publikováno v: Current Topics in Microbiology and Immunology ISBN: 9783642626678

The development of efficient lentivectors brings about exciting possibilities for novel therapeutic interventions. Still, as new biologicals intended to be used in the clinic, these vectors will have to comply with a complete set of requirements rega

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::6225998fc6f59c360559e1684a314e85
https://doi.org/10.1007/978-3-642-56114-6_6

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