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

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Author(s)

Abstract

<p>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 <i>in vivo</i> gene transfer to target tissues or administration of <i>ex vivo</i> 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.</p>

Journal

  • Chemical and Pharmaceutical Bulletin

    Chemical and Pharmaceutical Bulletin 66(3), 217-224, 2018

    The Pharmaceutical Society of Japan

Codes

  • NII Article ID (NAID)
    130006407125
  • NII NACSIS-CAT ID (NCID)
    AA00602100
  • Text Lang
    ENG
  • ISSN
    0009-2363
  • NDL Article ID
    028853931
  • NDL Call No.
    Z53-D167
  • Data Source
    NDL  J-STAGE 
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