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Summary for Patent: 7,998,734
|Title:||Methods and compositions for use in gene therapy for treatment of hemophilia|
|Abstract:||The invention includes a composition comprising a recombinant adeno-associated viral vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding Factor IX and accompanying 5\' and 3\' untranslated regions and a transcription termination.|
|Inventor(s):||High; Katherine A. (Merion, PA), Herzog; Roland W. (Glenolden, PA)|
|Assignee:||The Children\'s Hospital of Philadelphia (Philadelphia, PA)|
|Patent Claims:||1. A pharmaceutical composition comprising a virus, said virus comprising a recombinant adeno-associated virus vector comprising at least two adeno-associated virus inverted
terminal repeats, a promoter/regulatory sequence, isolated DNA encoding Factor IX and accompanying 5' and 3' untranslated regions and a transcription termination sequence, wherein said isolated DNA encoding Factor IX comprises a mutation encoding an
alanine residue in place of lysine in the fifth amino acid position from the beginning of mature Factor IX.
2. The composition of claim 1, further comprising a portion of intron 1 of the Factor IX gene.
3. The composition of claim 2, wherein said portion of intron 1 of a Factor IX gene is from about 0.3 kb to about 1.7 kb.
4. The composition of claim 1 further comprising a pharmaceutically acceptable carrier.
5. The composition of claim 1, wherein said promoter/regulatory sequence comprises the cytomegalovirus immediate early promoter/enhancer.
6. The composition of claim 1, wherein said transcription termination signal is the SV40 transcription termination signal.
7. A kit comprising the pharmaceutical composition of claim 1.
8. The composition of claim 1 wherein said Factor IX is human Factor IX.
|Applicant||Tradename||Biologic Ingredient||Dosage Form||BLA||Approval Date||Patent No.||Expiredate|
|Merck Sharp & Dohme Corp.||INTRON A||interferon alfa-2b||For Injection||103132||1986-06-04||⤷ Try it Free||2017-03-14|
|Merck Sharp & Dohme Corp.||INTRON A||interferon alfa-2b||For Injection||103132||⤷ Try it Free||2017-03-14|
|>Applicant||>Tradename||>Biologic Ingredient||>Dosage Form||>BLA||>Approval Date||>Patent No.||>Expiredate|
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