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Intrathymic AAV gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells.

08:00 EDT 9th September 2019 | BioPortfolio

Summary of "Intrathymic AAV gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells."

Patients with T cell immunodeficiencies are generally treated by allogeneic hematopoietic stem cell transplantation but alternatives are needed for patients without matched donors. An innovative intrathymic (IT) gene therapy approach, directly targeting the thymus, may improve outcome.

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This article was published in the following journal.

Name: The Journal of allergy and clinical immunology
ISSN: 1097-6825
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Medical and Biotech [MESH] Definitions

The introduction of functional (usually cloned) GENES into cells. A variety of techniques and naturally occurring processes are used for the gene transfer such as cell hybridization, LIPOSOMES or microcell-mediated gene transfer, ELECTROPORATION, chromosome-mediated gene transfer, TRANSFECTION, and GENETIC TRANSDUCTION. Gene transfer may result in genetically transformed cells and individual organisms.

The small RNA molecules, 73-80 nucleotides long, that function during translation (TRANSLATION, GENETIC) to align AMINO ACIDS at the RIBOSOMES in a sequence determined by the mRNA (RNA, MESSENGER). There are about 30 different transfer RNAs. Each recognizes a specific CODON set on the mRNA through its own ANTICODON and as aminoacyl tRNAs (RNA, TRANSFER, AMINO ACYL), each carries a specific amino acid to the ribosome to add to the elongating peptide chains.

Mutation process that restores the wild-type PHENOTYPE in an organism possessing a mutationally altered GENOTYPE. The second "suppressor" mutation may be on a different gene, on the same gene but located at a distance from the site of the primary mutation, or in extrachromosomal genes (EXTRACHROMOSOMAL INHERITANCE).

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The transfer of bacterial DNA by phages from an infected bacterium to another bacterium. This also refers to the transfer of genes into eukaryotic cells by viruses. This naturally occurring process is routinely employed as a GENE TRANSFER TECHNIQUE.

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