Modeling muscular diseases by patient-derived iPS cells

Modeling neurological diseases using patient-derived induced pluripotent stem cells.

Reprogramming of somatic cells to an embryonic-like state has dramatically changed the landscape of stem cell research. Although still in its formative stages, the field of induced pluripotent stem cells (iPSCs) has the potential to advance the study of neurodegenerative and neurodevelopmental disorders at the molecular and cellular levels. The iPSC technology could be employed to establish in ...

Modeling retinal degenerative diseases with human iPS-derived cells: current status and future implications.

David M Gamm, Department of Ophthalmology and Visual Sciences, McPherson Eye Research Institute, Waisman Center Stem Cell Research Program, University of Wisconsin School of Medicine and Public Health, Waisman Center, Room T609, 1500 Highland Ave, Madison, WI 53705, USA M Joseph Phillips, and Waisman Center Stem Cell Research Program, University of Wisconsin School of Medicine and Public Health...

Patient-derived cells modeling pediatric glioma

Patient-Specific iPSC-Derived RPE for Modeling of Retinal Diseases

Inherited retinal diseases, such as age-related macular degeneration and retinitis pigmentosa, are the leading cause of blindness in the developed world. Currently, treatments for these conditions are limited. Recently, considerable attention has been given to the possibility of using patient-specific induced pluripotent stem cells (iPSCs) as a treatment for these conditions. iPSCs reprogrammed...

Complete Genetic Correction of iPS Cells From Duchenne Muscular Dystrophy

Human artificial chromosome (HAC) has several advantages as a gene therapy vector, including stable episomal maintenance that avoids insertional mutations and the ability to carry large gene inserts including the regulatory elements. Induced pluripotent stem (iPS) cells have great potential for gene therapy, as such cells can be generated from the individual's own tissues, and when reintroduced...