Drug Delivery System

Feature articles “Development of new technologies toward the clinical trials of gene therapy” Editor : Mahito Nakanishi

Development of novel RNA vectors capable of stable and persistent gene expression

Mahito Nakanishi

2007 Volume 22 Issue 6 Pages 620-627
Published: 2007
Released on J-STAGE: February 18, 2008

DOIhttps://doi.org/10.2745/dds.22.620

JOURNAL FREE ACCESS

Show abstractHide abstract

Effective treatment of hereditary metabolic diseases requires long-term expression of therapeutic genes without chromosomal insertion. In this review, I describes the characteristics and application of unique RNA vectors capable of persistent gene expression with defect in interferon induction.

View full abstract

Download PDF (444K)
Selective gene delivery into target cells by fiber-modified adenovirus

Kazunori Kato, Hirofumi Hamada

2007 Volume 22 Issue 6 Pages 628-635
Published: 2007
Released on J-STAGE: February 18, 2008

DOIhttps://doi.org/10.2745/dds.22.628

JOURNAL FREE ACCESS

Show abstractHide abstract

We focused on the function of fiber-modified adenovirus (Adv-FZ33) that contains a synthetic 33-amino acid derived from protein A. We established the comprehensive procedure for screening antibodies that increase transduction efficiency by binding Adv-FZ33 with various human cancers.

View full abstract

Download PDF (594K)
Development of vaccine technology using Sendai virus vector

Makoto Inoue

2007 Volume 22 Issue 6 Pages 636-642
Published: 2007
Released on J-STAGE: February 18, 2008

DOIhttps://doi.org/10.2745/dds.22.636

JOURNAL FREE ACCESS

Show abstractHide abstract

The cytoplasmic RNA vector would be promising for use in gene vaccines to large population of patients because of its important genotoxicity-free nature. The candidate intranasal gene vaccines have been developed using Sendai virus(SeV) vector for both infectious and chronic diseases.

View full abstract

Download PDF (498K)
Gene therapy using AAV vectors

Keiya Ozawa

2007 Volume 22 Issue 6 Pages 643-650
Published: 2007
Released on J-STAGE: February 18, 2008

DOIhttps://doi.org/10.2745/dds.22.643

JOURNAL FREE ACCESS

Show abstractHide abstract

AAV(adeno-associated virus) vectors are considered to be promising gene-delivery vehicles for gene therapy, because they are derived from non-pathogenic virus, efficiently transduce non-dividing cells, and cause long-term gene expression. Appropriate AAV serotypes are utilized depending on the type of target cells. Among various neurological disorders, Parkinson's disease is one of the most promising candidates of gene therapy. On the other hand, intramuscular injection of AAV vectors is appropriate to protein-supplement gene therapy. Monogenic diseases such as hemophilia are suitable candidates.

View full abstract

Download PDF (471K)
Quality control and safety of human cell therapy products

Teruhide Yamaguchi, Eriko Uchida

2007 Volume 22 Issue 6 Pages 651-659
Published: 2007
Released on J-STAGE: February 18, 2008

DOIhttps://doi.org/10.2745/dds.22.651

JOURNAL FREE ACCESS

Show abstractHide abstract

ICH gene therapy discussion group has been expected to exchange information with the following objectives:monitoring emerging scientific issues, developing new ways of communication to ensure that the outcomes of ICH are well understood and widely disseminated, proactively setting out principles that may have a beneficial impact on harmonizing regulation of gene therapy products. The group has been discussing and published the concept papers about reference materials for gene therapy vectors, virus/vector shedding, evaluation of risk of X-SCID gene therapy, oncolytic virus products, and risk of germline integration of gene therapy products.

View full abstract

Download PDF (557K)

Register with J-STAGE for free!