Usage of U7 snRNA in gene therapy of hemoglobin S disorder--is it feasible?/ Hemoglobin S hastaliginin gen tedavisinde U7 snRNA kullanimi uygun mu?
Here, the author performs a basic bioinformatic analysis to assess the effect of co-expression between nucleic acid sequence for human Hb S beta globin chain and U7.623. To answer the raised question, a gene ontology technique was applied. The database PubMed was used for data mining of the nucleic acid sequence for human beta globin chain, and then the mutation beta 6 was experimentally done to obtain the primary sequence in Hb S disorder. In addition, the modified U7 snRNA (U7.623) was also searched and used for further study. Later, the author performed prediction of molecular function and biological process for the combination between nucleic acid sequence for human Hb S beta globin chain and U7.623 using a novel gene ontology prediction tool, GoFigure, which accepts an input DNA or protein sequence, and uses BLAST to identify homologous sequences in gene ontology databases . As a result, the molecular function and biological process in the co-expression between nucleic acid sequence for human Hb S beta globin chain and U7.623 was predicted as oxygen transporter activity for molecular function and oxygen transport for biological process, indicating complete recovery.
Gene therapy is a new therapeutic focus in modern medicine for many genetic diseases including hemoglobinopathy . Concerning thalassemia, modified U7 snRNA  is a widely documented SnRNA for therapy for beta globin gene defect. Here, the author assessed the effect of co-expression between nucleic acid sequence for human Hb S beta globin chain and U7.623 by the standard published technique by Wiwanitkit . According to this study, the gene ontological results showed that full recovery of hemoglobin in both molecular function and biological process can be achieved. Indeed, U7.623 antisense is already confirmed for its effectiveness in repairing gene therapy for several beta-thalassemia mutations [5-8]. However, there is a limited knowledge on Hb S disorder. This work is intended to extend our knowledge, and it implies the usefulness of this antisense approach to correct splicing defects in Hb S disorder. However, this is only a prediction that needs to be confirmed by in vitro analysis.
Received: December 13, 2007 Accepted: June 10, 2009
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Address for Correspondence: Dr. Viroj Wiwanitkit, Wiwanitkit House, Bangkhae, 10160 Bangkok, Thailand Phone: 662-2564136 E-mail: email@example.com
Wiwanitkit House, Bangkhae, 10160 Bangkok , Thailand
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|Title Annotation:||small nuclear deoxyribonucleic acid|
|Publication:||Turkish Journal of Hematology|
|Date:||Sep 1, 2009|
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