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Immunomodulatory gene therapy in lysosomal storage disorders.

Publication ,  Journal Article
Koeberl, DD; Kishnani, PS
Published in: Curr Gene Ther
December 2009

Significant advances in therapy for lysosomal storage disorders have occurred with an accelerating pace over the past decade. Although enzyme replacement therapy has improved the outcome of lysosomal storage disorders, antibody responses have occurred and sometimes prevented efficacy, especially in cross-reacting immune material negative patients with Pompe disease. Preclinical gene therapy experiments have revealed the relevance of immune responses to long-term efficacy. The choice of regulatory cassette played a critical role in evading humoral and cellular immune responses to gene therapy in knockout mouse models, at least in adult animals. Liver-specific regulatory cassettes prevented antibody formation and enhanced the efficacy of gene therapy. Regulatory T cells prevented transgene directed immune responses, as shown by adoptive transfer of antigen-specific immune tolerance to enzyme therapy. Immunomodulatory gene therapy with a very low vector dose could enhance the efficacy of enzyme therapy in Pompe disease and other lysosomal storage disorders.

Duke Scholars

Published In

Curr Gene Ther

DOI

EISSN

1875-5631

Publication Date

December 2009

Volume

9

Issue

6

Start / End Page

503 / 510

Location

United Arab Emirates

Related Subject Headings

  • Transgenes
  • T-Lymphocytes, Regulatory
  • Lysosomal Storage Diseases
  • Humans
  • Genetic Therapy
  • Biotechnology
  • Antigens, CD
  • 42 Health sciences
  • 32 Biomedical and clinical sciences
  • 31 Biological sciences
 

Citation

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Koeberl, D. D., & Kishnani, P. S. (2009). Immunomodulatory gene therapy in lysosomal storage disorders. Curr Gene Ther, 9(6), 503–510. https://doi.org/10.2174/156652309790031094
Koeberl, Dwight D., and Priya S. Kishnani. “Immunomodulatory gene therapy in lysosomal storage disorders.Curr Gene Ther 9, no. 6 (December 2009): 503–10. https://doi.org/10.2174/156652309790031094.
Koeberl DD, Kishnani PS. Immunomodulatory gene therapy in lysosomal storage disorders. Curr Gene Ther. 2009 Dec;9(6):503–10.
Koeberl, Dwight D., and Priya S. Kishnani. “Immunomodulatory gene therapy in lysosomal storage disorders.Curr Gene Ther, vol. 9, no. 6, Dec. 2009, pp. 503–10. Pubmed, doi:10.2174/156652309790031094.
Koeberl DD, Kishnani PS. Immunomodulatory gene therapy in lysosomal storage disorders. Curr Gene Ther. 2009 Dec;9(6):503–510.

Published In

Curr Gene Ther

DOI

EISSN

1875-5631

Publication Date

December 2009

Volume

9

Issue

6

Start / End Page

503 / 510

Location

United Arab Emirates

Related Subject Headings

  • Transgenes
  • T-Lymphocytes, Regulatory
  • Lysosomal Storage Diseases
  • Humans
  • Genetic Therapy
  • Biotechnology
  • Antigens, CD
  • 42 Health sciences
  • 32 Biomedical and clinical sciences
  • 31 Biological sciences