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Factor VIII adeno-associated virus vectors
专利权人:
BIOMARIN PHARMACEUTICAL; INC.
发明人:
PETER C. COLOSI
申请号:
ARP140103423
公开号:
AR097657A1
申请日:
2014.09.12
申请国别(地区):
AR
年份:
2016
代理人:
摘要:
The application provides delivery of adeno-associated virus (AAV) factor VIII (FVIII), which include AAV vectors of FVIII producing factor VIII polypeptide Functional AAV vectors with high expression of FVIII activity.Claim 2: The AAV vector of FVIII in the claim 1 wherein the Nucleic Acid comprising a Nucleotide sequence selected from the group consisting of: i) The proto 1 sequence in SEQ ID no. 1 set, (ii) the sequence proto 1S established in SEQ ID no. 2, (iii) the P sequence Broken 25 established in SEQ ID no. 3, (iv) The proto 3S Sequence established in SEQ ID no. 4(V) The proto 4 Sequence established in SEQ ID no. 5, (VI) sequence in SEQ ID no. 5 established proto proto 6, VII) sequence in SEQ ID 6 established no. 7, (viii) sequence in SEQ ID no. 7 established proto IX) 8, the sequence Construct 100atg established in SEQ ID no. 9 The sequence X, CO) Nstruct 100atg BGH Poly to established in SEQ ID no 10(XI) the sequence Construct 100atg Short BGH Poly to established in SEQ ID no. 11, the sequence Construct 103atg established in SEQ ID no. 12, XIII) the sequence Construct 103atg Short BGH Poly to established in SEQ ID no. 13, XIV) sequence to construct 105atg BGH Poly established in SEQ ID n No. 14, XV) established dc172atg FVIII Construct sequence in SEQ ID no. 15(XVI) the sequence Construct dc172 ATG FVIII hAAT established in SEQ ID no. 16, 17th) the sequence Construct dc172 2xhcr ATG FVIII established in SEQ ID no. 17, 18th century) the sequence Construct dc172 2xhcr ATG FVIII hAAT established in SEQ ID no. 18, 19th) the sequence Construct 2x FVIII serpinahaat ATG Established in SEQ ID no. 19XX) serpinahaat ATG FVIII Sequence Construct 2x 2x Enhancer micro globulin established in SEQ ID no. 20, XXI) sequence 100atg Enhancer Construct Short Poly to 2x micro globulin established in SEQ ID no. 21, XXII) the sequence Construct factor VIII - bmn001 established in SEQ ID n No. 22, XXIII), the sequence Construct factor VIII - bmn002 established in SEQ ID no. 23(XX
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