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Gene Therapy



Reports outline gene therapy study results from Mahidol University



August 14th, 2008

"A beta-thalassaemia mutation occurring from insertion of a duplicated 22-bp intron/exon junction of the beta-globin gene has been characterised," scientists writing in the journal Annals of Hematology report.

"The repeated 22-bp insertion causes duplication of a 3' splice site at IVSI/exon 2 junction," wrote S. Svasti and colleagues, Mahidol University.

The researchers concluded: "Reverse transcription-polymerase chain reaction showed that the proximal 3' splice site present in the duplicated gene is used, leading to a frameshift and a premature chain termination at codon 37. beta-Globin messenger ribonucleic acid (mRNA) transcribed from the...


Source: Gene Therapy Weekly (2008-08-14)

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