-
- OTSU Makoto
- Division of Stem Cell Processing/Stem Cell Bank, Institute of Medical Science, University of Tokyo
Bibliographic Information
- Other Title
-
- 患者由来iPS細胞を用いた遺伝子治療研究
- カンジャ ユライ iPS サイボウ オ モチイタ イデンシ チリョウ ケンキュウ
Search this article
Abstract
Gene therapy targeting hematopoietic stem cells (HSC) can now be recognized as a curative treatment option for patients with genetic disorders, including primary immunodeficiency (PID) diseases. Despite an increasing number of successfully treated cases, the therapeutic benefits still vary considerably among trials. To further optimize HSC gene therapy, it is hoped that a research model system capable of faithful recapitulation of the disease phenotypes can be established. Recently, a new model system that may meet this goal has become a reality; that is, patient-derived induced pluripotent stem cells (iPSCs). iPSCs are useful for modeling genetic disorders, because of their potential to differentiate into various types of somatic cells while retaining the specific genetic mutations. They are also susceptible to genetic manipulation in vitro, thus enabling pre-clinical assessment of candidate treatment strategies for their performance. This article introduces a proof that patient-derived iPSCs represent an invaluable tool for modelling genetic diseases such as PIDs, and also provide an indispensable research model usable for the development of ideal therapeutic modalities.
Journal
-
- Rinsho Ketsueki
-
Rinsho Ketsueki 56 (8), 1016-1024, 2015
The Japanese Society of Hematology
- Tweet
Details 詳細情報について
-
- CRID
- 1390282680011696768
-
- NII Article ID
- 130005096283
-
- NII Book ID
- AN00252940
-
- ISSN
- 18820824
- 04851439
-
- NDL BIB ID
- 026704106
-
- PubMed
- 26345561
-
- Text Lang
- ja
-
- Data Source
-
- JaLC
- NDL
- PubMed
- CiNii Articles
-
- Abstract License Flag
- Disallowed