Artemis splice defects cause atypical SCID and can be restored in vitro by an antisense oligonucleotide
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چکیده
منابع مشابه
Mutagenesis by an antisense oligonucleotide and its degradation product.
The European Medicines Agency has expressed concern regarding (1) the potential for antisense oligonucleotide (ASO) therapeutics to induce sequence-specific mutation at genomic DNA and (2) the capability of ASO degradation products (nucleotide analogues) to incorporate into newly synthesized genomic DNA via DNA polymerase and cause mutation if base pairing occurs with reduced fidelity. Treating...
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A subgroup of severe combined immunodeficiencies (SCID) is characterized by lack of T and B cells and is caused by defects in genes required for T- and B-cell receptor gene rearrangement. Several of these genes are also involved in nonhomologous end joining of DNA double-strand break repair, the largest subgroup consisting of patients with T(-)B(-)NK(+)SCID due to DCLRE1C/ARTEMIS defects. We po...
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BACKGROUND Current vaccination strategies and antiviral drugs only provide limited protection against influenza virus infection. In this study, we investigated the use of a novel antisense oligonucleotide (named IV-AS), which is specific for the 5'-terminal conserved sequence found in all eight viral RNA segments of influenza A virus. METHODS The activity of IV-AS was monitored both in vitro,...
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ژورنال
عنوان ژورنال: Genes & Immunity
سال: 2011
ISSN: 1466-4879,1476-5470
DOI: 10.1038/gene.2011.16