Abstract

Stem-cell-based therapies are considered to be promising and innovative but complex approaches. Induced pluripotent stem cells (iPSCs) combine the advantages of adult stem cells with the hitherto unique characteristics of embryonic stem cells (ESCs). Major progress has already been achieved with regard to reprogramming technology, but also regarding targeted genome editing and scalable expansion and differentiation of iPSCs and ESCs, in some cases yielding highly enriched preparations of well-defined cell lineages at clinically required dimensions. It is noteworthy, however, that for many applications critical requirements such as the targeted specification into distinct cellular subpopulations and a proper cell maturation remain to be achieved. Moreover, current hurdles such as low survival rates and insufficient functional integration of cellular transplants remain to be overcome. Nevertheless, PSC technologies obviously have come of age and matured to a stage where various clinical applications of PSC-based cellular therapies have been initiated and are conducted.

Highlights

  • Modern medical technologies with the countless number of available drugs, new diagnostic procedures, and therapeutic applications including complex surgical interventions greatly contributed to a substantially increased life expectancy in industrialized countries

  • Treatment of dry age-related macular degeneration disease with retinal pigment epithelium (RPE) derived from human embryonic stem cells (ESCs)

  • These include, for instance, life-threatening arrhythmia after transplantation of pluripotent stem cells (PSCs)-derived cardiomyocytes, infection risks caused by the application procedure, excessive inflammation due to donor cell death, and immune reactions against transgenic grafts or graft vs. host reactions in case of hematopoietic transplants

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Summary

Ulrich Martin*

Major progress has already been achieved with regard to reprogramming technology, and regarding targeted genome editing and scalable expansion and differentiation of iPSCs and ESCs, in some cases yielding highly enriched preparations of well-defined cell lineages at clinically required dimensions. It is noteworthy, that for many applications critical requirements such as the targeted specification into distinct cellular subpopulations and a proper cell maturation remain to be achieved.

INTRODUCTION
Stem cell therapy for outer retinal degenerations
Observational study
Neurological disorders
Thalassemia treatment based on the stem cell technology
GENERAL REGULATORY REQUIREMENTS
PSCs for Treatment of Ophthalmic Diseases
PSCs for Treatment of Neurological Disorders
PSCs for Treatment of Diabetes
PSCs for Clinical Heart Repair
Full Text
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