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Garcia-Perez Et Al., 2020 by jmiao0901 is a document available to read on EtoBox.
What is Garcia-Perez Et Al., 2020 about?
The study presents successful preclinical development of gene therapy for RAG1-deficient severe combined immunodeficiency (SCID) using lentiviral vectors that drive codon-optimized RAG1 expression. The results indicate that higher RAG1 expression leads to effective immune reconstitution in a mouse model, supporting the potential for clinical trials. This approach offers a promising alternative for SCID patients lacking suitable bone marrow donors.
- Author
- jmiao0901
- Language
- EN