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A Novel Approach to the Treatment of Plasma Protein Deficiency: Ex Vivo-Manipulated Adipocytes for Sustained Secretion of Therapeutic Proteins.

Authors
  • Kuroda, Masayuki1
  • Saito, Yasushi2
  • Aso, Masayuki3
  • Yokote, Koutaro4
  • 1 Center for Advanced Medicine, Chiba University Hospital, Chiba University.
  • 2 Hospital Bureau.
  • 3 CellGenTech, Inc.
  • 4 Clinical Cell Biology and Medicine, Chiba University Graduate School of Medicine, Chiba University.
Type
Published Article
Journal
Chemical and Pharmaceutical Bulletin
Publisher
Pharmaceutical Society of Japan
Publication Date
Jan 01, 2018
Volume
66
Issue
3
Pages
217–224
Identifiers
DOI: 10.1248/cpb.c17-00786
PMID: 29491255
Source
Medline
Keywords
License
Unknown

Abstract

Despite the critical need for lifelong treatment of inherited and genetic diseases, there are no developmental efforts for most such diseases due to their rarity. Recent progress in gene therapy, including the approvals of two products (Glybera and Strimvelis) that may provide patients with sustained effects, has shed light on the development of gene therapy products. Most gene therapy products are based on either adeno-associated virus-mediated in vivo gene transfer to target tissues or administration of ex vivo gene-transduced hematopoietic cells. In such circumstances, there is room for different approaches to provide clinicians with other therapeutic options through a variety of principles based on studies not only to gain an understanding of the pathological mechanisms of diseases, but also to understand the physiological functions of target tissues and cells. In this review, we summarize recent progress in gene therapy-mediated enzyme replacement and introduce a different approach using adipocytes to enable lifelong treatment for intractable plasma protein deficiencies.

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