Abstract:
:A chimeric RNA/DNA oligonucleotide was constructed to induce a sequence mutation in the rat factor IX gene, resulting in prolonged coagulation. Oligonucleotides were targeted to hepatocytes in cell culture or in vivo by intravenous injection. Nucleotide conversion was both site-specific and dose-dependent. The mutated gene was associated in vivo with significantly reduced factor IX coagulant activity and a marked prolongation of the activated partial thromboplastin time. The results demonstrate that single base-pair alterations can be introduced in hepatocytes in situ by RNA/DNA oligonucleotides, suggesting a potentially powerful strategy for hepatic gene repair without the use of viral vectors.
journal_name
Nat Medjournal_title
Nature medicineauthors
Kren BT,Bandyopadhyay P,Steer CJdoi
10.1038/nm0398-285subject
Has Abstractpub_date
1998-03-01 00:00:00pages
285-90issue
3eissn
1078-8956issn
1546-170Xjournal_volume
4pub_type
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