Article
Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome.
The Journal of allergy and clinical immunology - 1 Apr 2014
van Til Niek P, Sarwari Roya, Visser Trudi P, Hauer Julia, Lagresle-Peyrou Chantal, van der Velden Guus, Malshetty Vidyasagar, Cortes Patricia, Jollet Arnaud, Danos Olivier, Cassani Barbara, Zhang Fang, Thrasher Adrian J, Fontana Elena, Poliani Pietro L, Cavazzana Marina, Verstegen Monique M A, Villa Anna, Wagemaker Gerard
Abstract excerpt
BACKGROUND: Recombination-activating gene 1 (RAG1) deficiency results in severe combined immunodeficiency (SCID) caused by a complete lack of T and B lymphocytes. If untreated, patients succumb to recurrent infections. OBJECTIVES: We sought to develop lentiviral gene therapy for RAG1-induced SCID and to test its safety. METHODS: Constructs containing the viral spleen-focus-forming virus (SF), ubiquitous...
Topics
- Animals
- Autoimmunity
- Bone Marrow Cells
- Disease Models, Animal
- Female
- Gene Dosage
- Gene Expression
- Genetic Therapy
- Genetic Vectors