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Lysosomal Hydrolases01:22

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Gene therapy for lysosomal storage disorders.

Nelson S Yew1, Seng H Cheng2

  • 1Genzyme, Sanofi Company, 49 New York Avenue, Framingham, MA 01701-9322, USA. nelson.yew@genzyme.com

Pediatric Endocrinology Reviews : PER
|January 2, 2014
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Summary

Gene transfer using adeno-associated virus (AAV) and lentiviral vectors shows promise for treating lysosomal storage diseases (LSDs). These advanced therapies demonstrate effective gene delivery to both central and peripheral tissues in mouse models.

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Area of Science:

  • Biomedical research
  • Genetics
  • Molecular biology

Background:

  • Lysosomal storage diseases (LSDs) are inherited single-gene disorders.
  • Gene transfer is a promising therapeutic strategy for LSDs.
  • Significant advancements have been made in gene delivery technologies.

Purpose of the Study:

  • To review recent progress in using gene transfer for LSDs.
  • To highlight the efficacy of adeno-associated virus (AAV) and lentiviral vectors.
  • To discuss challenges in translating these therapies to human patients.

Main Methods:

  • Utilizing recombinant adeno-associated virus (AAV) vectors.
  • Employing lentiviral vectors for gene delivery.
  • Evaluating gene expression in mouse models of LSDs.

Main Results:

  • AAV and lentiviral vectors enable widespread, robust, and sustained gene expression.
  • Successful gene delivery to both peripheral and central nervous systems achieved.
  • Demonstrated therapeutic potential in various LSD mouse models.

Conclusions:

  • Gene transfer with AAV and lentiviral vectors is a viable approach for LSD treatment.
  • Further research is needed to overcome challenges for human clinical application.
  • Translational studies are crucial to bridge the gap between mouse models and human patients.