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Adeno-associated viral vectors: background and technical aspects.

Publication ,  Journal Article
Snoeck, HW; Tao, W; Klotman, ME
Published in: Exp Nephrol
1997

There are several obstacles that prevent the successful clinical application of gene therapy. Some of these challenges are unique to the particular disease and organ that is being targeted. Desirable characteristics of approaches aimed at delivery of a therapeutic gene to the kidney ideally will require a vector that is safe, that efficiently transduces nondividing cells, and that can lead to long-term gene expression. Viral vectors that are derived from the small replication-deficient parvovirus, adeno-associated virus, offer many potential advantages. The wild-type virus is nonpathogenic and can site specifically integrate at a single location on chromosome 19, a process that offers the hope that this characteristic could be engineered into recombinant vectors as well. Recombinant adeno-associated virus can also efficiently integrate into the host genome, can transduce nondividing cells, and does not induce an immune response which destroys the transduced cells. Efforts focused both on gaining a more complete understanding of the virus life cycle as well on the efficient production of high-titer virus should bring this vector closer to clinical application.

Duke Scholars

Published In

Exp Nephrol

ISSN

1018-7782

Publication Date

1997

Volume

5

Issue

6

Start / End Page

514 / 520

Location

Switzerland

Related Subject Headings

  • Urology & Nephrology
  • Humans
  • Genetic Vectors
  • Gene Transfer Techniques
  • Dependovirus
  • DNA, Recombinant
  • 3202 Clinical sciences
  • 1116 Medical Physiology
  • 1103 Clinical Sciences
 

Citation

APA
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ICMJE
MLA
NLM
Snoeck, H. W., Tao, W., & Klotman, M. E. (1997). Adeno-associated viral vectors: background and technical aspects. Exp Nephrol, 5(6), 514–520.
Snoeck, H. W., W. Tao, and M. E. Klotman. “Adeno-associated viral vectors: background and technical aspects.Exp Nephrol 5, no. 6 (1997): 514–20.
Snoeck HW, Tao W, Klotman ME. Adeno-associated viral vectors: background and technical aspects. Exp Nephrol. 1997;5(6):514–20.
Snoeck, H. W., et al. “Adeno-associated viral vectors: background and technical aspects.Exp Nephrol, vol. 5, no. 6, 1997, pp. 514–20.
Snoeck HW, Tao W, Klotman ME. Adeno-associated viral vectors: background and technical aspects. Exp Nephrol. 1997;5(6):514–520.

Published In

Exp Nephrol

ISSN

1018-7782

Publication Date

1997

Volume

5

Issue

6

Start / End Page

514 / 520

Location

Switzerland

Related Subject Headings

  • Urology & Nephrology
  • Humans
  • Genetic Vectors
  • Gene Transfer Techniques
  • Dependovirus
  • DNA, Recombinant
  • 3202 Clinical sciences
  • 1116 Medical Physiology
  • 1103 Clinical Sciences