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Potential limits of AAV-based gene therapy with the use of new transgenes expressing factor IX fusion proteins.

Haemophilia. 2019-01; 
Le QuellecSandra, DaneAllison, EnjolrasNathalie, McIntoshJenny, RosalesCecilia, NegrierClaude, Nathwani
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Codon Optimization … vector containing 201 bp of the 3' end of humanly codon‐optimised hFIX (hFIXco) cDNA sequence followed by full linker and mature albumin cDNA sequences, flanked by EcoNI and NheI restriction sites were synthesized (GenScript Biotech Corp., Nanjing, China) and cloned … Get A Quote

摘要

The variety of treatment for haemophilia B (HB) has recently improved with the emergence of both AAV-based gene therapy and bioengineered human factor IX (hFIX) molecules with prolonged half-life due to fusion to either albumin (Alb) or immunoglobulin Fc fragment (Fc).

关键词

Fc fusion，adeno-associated virus，albumin fusion，factor IX，gene therapy，haemoph
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