At the Alzheimer’s Association International Conference this past July, Andrew Sproul, PhD, a NYSCF Postdoctoral Associate, presented NYSCF’s latest efforts to understand Alzheimer’s disease. They have generated relevant induced pluripotent stem (iPS) cell models from AD patients and well relatives that were then differentiated into cholinergic neurons. Other efforts in the generation of human iPS cell disease models include cortical neurons from Down’s syndrome patients and the creation of cell lines from ALS patients. These iPS lines reveal the underlying biology that animal models do not.
Dieter Egli, a NYSCF Senior Research Fellow, contributed to a study published in Molecular Medicine that provides direct evidence of sight-loss recovery using induced pluripotent stem (iPS) cell transplantation. In collaboration with Stephen Tsang, PhD, from Columbia University, Egli provided human retinal pigmented epithelium cells, derived from iPS cells. These cells were then grafted into mice genetically engineered to suffer from retinitis pigmentosa, a degenerative eye disease that causes vision impairment and blindness. Tests to measure visual function revealed improved sight in these mice. This autologous iPS cell graft could lead to clinical applications in human patients diagnosed with macular or retinal degeneration.
Dung-Fang Lee, PhD, a NYSCF – Druckenmiller Fellow Alumnus at Mount Sinai School of Medicine, has published a new study in the August edition of Cell Stem Cell that elucidates a molecular signal necessary to maintain self-renewal and pluripotency in embryonic stem cells (ESCs) and somatic cells. When regulatory molecule aurora kinase A was depleted, Dr. Lee and his team found inhibited gene expression in ESCs. This suggests the essential role aurora kinase A signaling plays in cell differentiation, self-renewal, and reprogramming.
Ting Chen, PhD, a NYSCF – Druckenmiller Fellowship Alumna at The Rockefeller University, has published work in the April issue of Nature that sheds new light on the mechanisms that govern stem cell renewal and regeneration. By using RNA screening techniques on stem cells derived from human hair follicles, Dr. Chen and her team have identified TBX1, a transcription factor that plays an important role in regulating tissue regeneration by acting as a gatekeeper for cell proliferation. By identifying and observing the behavior of transcription factors like TBX1, scientists may gain valuable insight into cell regeneration in the hopes of developing ways to control cell behavior to treat diseases such as skin cancer.
Marius Wernig, MD, a NYSCF – Robertson Investigator at Stanford University, released an update to previously published work confirming that mouse skin cells can bypass the stem cell stage and be converted directly into cells that become the three main parts of the nervous system, including the neural precursor cells that develop into neurons. In conjunction with the prior research, this new study indicates strongly that this new technique may be used to quickly generate large amounts of neural precursor cells for use in disease modeling and drug screening. In addition, Dr. Wernig and his team are hopeful that the technique may hold therapeutic potential to generate patient-specific cells to replace ones damaged by disease. This study was announced online on January 30th in the Proceedings of the National Academy of Sciences.