A Novel Approach to the Treatment of Plasma Protein Deficiency : Ex Vivo-Manipulated Adipocytes for Sustained Secretion of Therapeutic Proteins
-
- Kuroda Masayuki
- Center for Advanced Medicine, Chiba University Hospital, Chiba University
-
- Saito Yasushi
- Hospital Bureau
-
- Aso Masayuki
- CellGenTech, Inc.
-
- Yokote Koutaro
- Clinical Cell Biology and Medicine, Chiba University Graduate School of Medicine, Chiba University
書誌事項
- タイトル別名
-
- A Novel Approach to the Treatment of Plasma Protein Deficiency: <i>Ex Vivo</i>-Manipulated Adipocytes for Sustained Secretion of Therapeutic Proteins
この論文をさがす
抄録
<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 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.</p>
収録刊行物
-
- CHEMICAL & PHARMACEUTICAL BULLETIN
-
CHEMICAL & PHARMACEUTICAL BULLETIN 66 (3), 217-224, 2018
公益社団法人 日本薬学会
- Tweet
キーワード
詳細情報 詳細情報について
-
- CRID
- 1390001204178766208
-
- NII論文ID
- 130006407125
-
- NII書誌ID
- AA00602100
-
- ISSN
- 13475223
- 00092363
-
- NDL書誌ID
- 028853931
-
- PubMed
- 29491255
-
- 本文言語コード
- en
-
- データソース種別
-
- JaLC
- NDL
- Crossref
- PubMed
- CiNii Articles
- KAKEN
-
- 抄録ライセンスフラグ
- 使用不可