Patient-derived iPS cells as a tool for gene therapy research

[Rinshō ketsueki] The Japanese journal of clinical hematology
Makoto Otsu

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.

Related Concepts

Related Feeds

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Related Papers

Korean journal of pediatrics
Hoon-Chul Kang
[Rinshō ketsueki] The Japanese journal of clinical hematology
Mineo Kurokawa
Nihon Rinshō Men'eki Gakkai kaishi = Japanese journal of clinical immunology
Makoto Otsu
© 2021 Meta ULC. All rights reserved