J 2013

Pluripotent stem cells and gene therapy

ŠIMARA, Pavel, Jason MOTL and Dan KAUFMAN

Basic information

Original name

Pluripotent stem cells and gene therapy

Authors

ŠIMARA, Pavel (203 Czech Republic, guarantor, belonging to the institution), Jason MOTL (840 United States of America) and Dan KAUFMAN (840 United States of America)

Edition

Translational Research, 2013, 1931-5244

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

10601 Cell biology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

Impact factor

Impact factor: 4.044

RIV identification code

RIV/00216224:14330/13:00068030

Organization unit

Faculty of Informatics

UT WoS

000316837400008

Keywords in English

pluripotent stem cells; gene therapy

Tags

Změněno: 2/3/2018 10:11, Mgr. Pavel Šimara, Ph.D.

Abstract

V originále

Human pluripotent stem cells represent an accessible cell source for novel cell-based clinical research and therapies. With the realization of induced pluripotent stem cells (iPSCs), it is possible to produce almost any desired cell type from any patient's cells. Current developments in gene modification methods have opened the possibility for creating genetically corrected human iPSCs for certain genetic diseases that could be used later in autologous transplantation. Promising preclinical studies have demonstrated correction of disease-causing mutations in a number of hematological, neuronal, and muscular disorders. This review aims to summarize these recent advances with a focus on iPSC generation techniques, as well as gene modification methods. We will then further discuss some of the main obstacles remaining to be overcome before successful application of human pluripotent stem cell-based therapy arrives in the clinic and what the future of stem cell research may look like.

Links

MSM0021622430, plan (intention)
Name: Funkční a molekulární charakteristiky nádorových a normálních kmenových buněk - identifikace cílů pro nová terapeutika a terapeutické strategie
Investor: Ministry of Education, Youth and Sports of the CR, Functional and molecular characteristics of cancer and normal stem cells - identification of targets for novel therapeutics and therapeutic strategies