Clinical Trial: Development of iPS From Donated Somatic Cells of Patients With Neurological Diseases
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: Derivation of Induced Pluripotent Stem Cells From Somatic Cells Donated by Patients With Neurological Diseases for the Study of the Pathogenesis of the Disorders and Development of Novel Therapies
Brief Summary:
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5). The iPS cells seem to share many properties with human embryonic stem cells.
Induced pluripotent stem cells potentially may be useful in the future as an unlimited source of cells for transplantation.
The major goal of the project is to develop human iPS cells from cell cultures from skin biopsies or the patient's hair. The iPS cells will be developed primarily for modeling diseases and drug discovery as well as basic research, and for developing the technology that may eventually allow the use of iPS cells for future transplantation therapy. The iPS cells developed in the course of this application are not intended for use in transplantation therapy. Future development of iPS cells for clinical transplantation therapies will be subjected to the appropriate authorization by ethical and regulatory committees.
Detailed Summary:
The derivation of human iPS cells (1-5) open new avenues to model human diseases since it may now be possible to develop iPS cells from the fibroblasts or other somatic cells of patients with various conditions. These iPS cells may be directed to differentiate into the cells which are affected in specific conditions. Abnormalities in the development of the affected cells as well as altered survival or function of the cells may be studied. Thus iPS cells may serve as an invaluable model for the study of the pathogenesis of human diseases and may also serve for the development of new drugs, and high throughput screening of molecules for toxic or therapeutic effects.
In addition to the great potential of iPS cells for disease modelling and transplantation therapy, the cells may have broad applications in basic research in various areas such as reprogramming, basic development and others.
At present, it is still unclear whether the properties of iPS cells are identical to those of hESCs. Initial data suggest that human iPS cells are indeed similar to hESCs in their phenotype, epigenetic status of pluripotent self-specific genes, telomerase activity, gene expression profile and in their capability to differentiate into progeny of the three germ layers both in vitro and in vivo in teratomas (2, 3, 5). In the mouse system, directed differentiation of iPS cells into bone marrow repopulating hematopoietic stem cells and functional dopaminergic neurons was demonstrated (6, 7). However, incomplete silencing of the constitutive expression of the transcription factors that were used to induce reprogramming can probably interfere with differentiation (1). Further studies are required to confirm that the developmental potential and biological properties of iPS cells are identical to hESC.
Sponsor: Hadassah Medical Organization
Current Primary Outcome:
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Original Secondary Outcome:
Information By: Hadassah Medical Organization
Dates:
Date Received: April 2, 2009
Date Started: April 2009
Date Completion: December 2020
Last Updated: February 28, 2017
Last Verified: February 2017
