A factor VIII minigene comprising the truncated intron I of factor IX highly improves the in vitro production of factor VIII

Thrombosis and Haemostasis

Volumn 86, Issue 2, 2001, Pages 596-603

  Plantier, J L ^a   Rodriguez, M H ^a   Enjolras, N ^a   Attali, O ^a   Negrier C ^a  

^a INSERM   (France)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; COMPLEMENTARY DNA; RECOMBINANT BLOOD CLOTTING FACTOR 8;

ANIMAL CELL; ANIMAL TISSUE; ARTICLE; BIOSYNTHESIS; BLOOD CLOTTING PARAMETERS; CHINESE HAMSTER; CONTROLLED STUDY; DNA SEQUENCE; DRUG SYNTHESIS; GENE INSERTION; GENE THERAPY; GENETIC ANALYSIS; GENETIC ASSOCIATION; GENETIC TRANSCRIPTION; HAMSTER; HEMOPHILIA A; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VITRO STUDY; INTRON; NONHUMAN; OVARY CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; PROTEIN PROCESSING; REGULATORY MECHANISM; TRANSCRIPTION TERMINATION;

EID: 0034905074     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1055/s-0037-1616092     Document Type: Article

References (48)

1. Characterization of the human factor VIII gene
2. R.M.H. Molecular cloning of a cDNA encoding human antihaemophilic factor
3. Structure of human factor VIII
4. Molecular genetics of coagulation factor VIII gene and hemophilia A
5. Haemophilia A: Database of nucleotide substitutions, deletions, insertions and rearrangements of the factor VIII gene, second edition
6. Prions and haemophilia: Assessment of risk
7. Viral safety of plasma-derived factor VIII and IX concentrates
8. Gene therapy for the hemophilias
9. Expression of the blood-clotting factor-VIII cDNA is repressed by a transcriptional silencer located in its coding region
10. The human clotting factor VIII cDNA contains an autonomously replicating sequence consensus- and matrix attachment region-like sequence that binds a nuclear factor, represses heterologous gene expression, and mediates the transcriptional effects of sodium butyrate
11. Sequences within the coding regions of clotting factor VIII and CFTR block transcriptional elongation
12. Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production
13. A 110-amino acid region within the A1-domain of coagulation factor VIII inhibits secretion from mammalian cells
14. Mutations in the ER-Golgi intermediate compartment protein ERGIC-53 cause combined deficiency of coagulation factors V and VIII
15. Differential interaction of coagulation factor VIII and factor V with protein chaperones calnexin and calreticulin
16. Effects of von Willebrand factor coexpression on the synthesis and secretion of factor VIII in chinese hamster ovary cells
17. The light chain of factor VIII comprises a binding site for low density lipoprotein receptor-related protein
18. Role of the low density lipoprotein-related protein receptor in mediation of factor VIII catabolism
19. Involvement of low-density lipoprotein receptor-related protein (LRP) in the clearance of factor VIII in von Willebrand factor-deficient mice
20. Factor VIII and von Willebrand factor
21. Evaluation of an adenoviral vector encoding full length human factor VIII in hemophiliac mice
22. Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero
23. Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
24. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
25. The three in-frame ATG, clustered in the translation initiation sequence of human factor IX gene, are required for an optimal protein production
26. Characterization of recombinant human factor IX expressed in transgenic mice and in derived trans-immortalized hepatic cell lines
27. Role of intron I in expression of the human factor IX gene
28. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro
29. Construction of human factor IX expression vectors in retroviral vector frames optimized for muscle cells
30. Recognition of AUG and alternative initiator codons is augmented by G in position +4 but is not generally affected by the nucleotides in positions +5 and +6
31. Nucleotide sequence of the gene for human factor IX (antihemophilic factor B)
32. Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization
33. Synthesis, processing and secretion of recombinant human factor VIII expressed in mammalian cells
34. Post-translational requirements for functional factor V and factor VIII secretion in mammalian cells
35. Post-transcriptional modifications required for coagulation factor secretion and function
36. Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A
37. Mutagenesis of a potential immunoglobulin-binding protein-binding site enhances secretion of coagulation factor VIII
38. Characterization of a genetically engineered inactivation-resistant coagulation factor VIIIa
39. Introns increase transcriptional efficiency in transgenic mice
40. Comparison of intron-dependent and intron-independent gene expression
41. Splicing and formation of stable RNA
42. High-level tissue-specific expression of functional human factor VIII in mice
43. Order of intron removal during splicing of endogenous adenine phosphoribosyltransferase and dihydrofolate reductase pre-mRNA
44. Differential distribution of alternatively spliced somatostatin receptor 2 isoforms (sst2A and sst2B) in rat spinal cord
45. A splicing-dependent regulatory mechanism that detects translation signals
46. Specific commitment of different pre-mRNAs to splicing by single SR proteins
47. In vivo evidence that transcription and splicing are coordinated by a recruiting mechanism
48. ATP-dependent dissociation of non-disulfide-linked aggregates of coagulation factor VIII is a rate-limiting step for secretion