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Correction of the beta°39 mRNA translation by aminoglycosides

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Manal:
This is one of the intersting findings that was discussed by Prof Gambari too


--- Quote ---Correction of the beta°39 mRNA translation by aminoglycosides. In beta039-thalassemia the CAG (Gln) codon of beta-globin mRNA is mutated to the UAG stop codon, leading to premature translation termination and mRNA destabilization through nonsense-mediated mRNA decay (NMD). We have found that aminoglycosides are potent correctors of this mRNA alterations, inducing production of adult hemoglobin by erythroid cells from beta°39 homozygous thal patients. This was assessed by FACS analysis and HPLC. RT-PCR suggest that this treatment leads to increase of beta°39 mRNA stability.

In conclusion, the data obtained extend the concept that several drugs from natural word are expected to be proposed for the cure of beta-thalassemia. We firmly believe that this field will be exciting from the scientific point of view, but also represent a hope for several patients, whose survival will depend of the possible use of drugs rendering not necessary blood transfusion and chelation therapy.
--- End quote ---


manal

Zaini:
Thanks for the info Manal  :hugfriend

Zaini.

Sharmin:
Manal,

Thank you so much for this information :) 

Sharmin

mohamed:
any other info about that topic Mrs Manal?

Manal:
Actually, no, but i am very interested to know how studies are going in this field (mutations in m(RNA) whether the processing, translation, splicing,...) . I assume that if there are any would be in the theoratical stage.

manal

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