Blog Entry 1:
Hi, my name is Emma Friedenberg I’m a CIRM-SPARK Intern working at the Cedars-Sinai Medical Center in Los Angeles. I am working in the Governors Board of Regenerative Medicine on the 8th floor under Dr. Virginia Mattis in her stem cell lab. My initiative is to use human patient induced pluripotent stem cells (iPSCs) to generate Huntington’s disease “in a dish” model to enhance basic understanding of disease mechanisms in the hopes of developing effective treatments. This involves studying the use factors to directly convert iPSC-derived neural progenitors into medium spiny neurons. Huntington’s disease (HD) is a fatal genetic disorder with no cure that causes the progressive breakdown of nerve cells in the brain. Mutations occur in the Huntington (HTT) gene creating an elongated glutamine chain of CAG repeats. While there is normally less than 36 repeating CAG codons, an individual with HD has more than 36 glutamine residues (individuals with the adult-onset form of HD typically have 40 to 50 CAG repeats, while people with the juvenile form of the disorder have more than 60). In the lab, we have a strain with over 200 repeats. HD affects the whole brain, but certain areas are more vulnerable than others such as the striatum. Medium spiny neurons compose 95-99% of the striatum. It is my task to create a more time and cost-effective procedure to produce medium spiny neurons from iPSCs. So far, I have observed the passage of stem cells with cutting edge machinery, created cell slides for imagining on the Leica scope and analyzed the images on ImageJ – counting nuclei, medium spiny neurons, and mature neurons.