I just got work yesterday from Hong that our collaboration with Dr. Kole has finally paid off. I am the 5th author on our new paper.
RNA repair restores hemoglobin expression in IVS2–654 thalassemic mice.
PNAS January 27, 2009 vol. 106 no. 4 pp1205–1210
Repair of b-globin pre-mRNA rendered defective by a thalassemia causing
splicing mutation, IVS2–654, in intron 2 of the human
b-globin gene was accomplished in vivo in a mouse model of
IVS2–654 thalassemia. This was effected by a systemically delivered
splice-switching oligonucleotide (SSO), a morpholino oligomer
conjugated to an arginine-rich peptide. The SSO blocked the
aberrant splice site in the targeted pre-mRNA and forced the
splicing machinery to reselect existing correct splice sites. Repaired
b-globin mRNA restored significant amounts of hemoglobin in the
peripheral blood of the IVS2–654 mouse, improving the number
and quality of erythroid cells.
Now I need to get published from my current lab. Hopefully it won't be too much longer.
Was that English? :) Just kidding...I am very proud of you! Can't wait to hear of your next published paper. xo B
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