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Liver-restricted expression of the canine factor VIII gene facilitates prevention of inhibitor formation in factor VIII-deficient mice

The Journal of Gene Medicine

Sep 17, 2009

Akira IshiwataY Sakata

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PubMed

Abstract

Gene therapy for hemophilia A with adeno-associated virus (AAV) vectors involves difficulties in the efficient expression of factor VIII (FVIII) and in antibody formation against transgene-derived FVIII. AAV8 vectors carrying the canine B domain deleted FVIII (cFVIII) gene under the con...read more

Mentioned in this Paper

Neutralising Antibodies Analysis
Beta-Actin
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Decreased Immunologic Activity [PE]
Virus Diseases
Isoactin
Adeno-Associated Virus
Hepatic
Promoter
Antibodies, Neutralizing
Paper Details
References
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  • Citations19
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  • References34
  • Citations19
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Liver-restricted expression of the canine factor VIII gene facilitates prevention of inhibitor formation in factor VIII-deficient mice

The Journal of Gene Medicine

Sep 17, 2009

Akira IshiwataY Sakata

PMID: 19757487

DOI: 10.1002/jgm.1391

Abstract

Gene therapy for hemophilia A with adeno-associated virus (AAV) vectors involves difficulties in the efficient expression of factor VIII (FVIII) and in antibody formation against transgene-derived FVIII. AAV8 vectors carrying the canine B domain deleted FVIII (cFVIII) gene under the con...read more

Mentioned in this Paper

Neutralising Antibodies Analysis
Beta-Actin
Shuttle Vectors
Decreased Immunologic Activity [PE]
Virus Diseases
Isoactin
Adeno-Associated Virus
Hepatic
Promoter
Antibodies, Neutralizing

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Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

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Paper Details
References
  • References34
  • Citations19
1234
  • References34
  • Citations19
12

Download from

Publisher
PubMed
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