The Friedman Brain Institute

Stem Cells

The first human embryonic stem cell lines were grown by Dr. James Thomson in Wisconsin in 1998.  These cells had the ability to make every cell type in the body, an unlimited source of material, but scientists couldn’t yet use them to study disease.

In 2006, Dr. Shinya Yamanaka, was trying to understand what gave embryonic stem cells their special ability to differentiate into all other cell types.  He narrowed it down to just four genes, a discovery for which he was awarded the 2012 Nobel Prize.

As a result, scientists around the world have gained the ability to make stem cells from every person on the planet.  In doing so, by making stem cells from anyone, they can make stem cells with any disease, and from these, make the cell types affected by any disease, and then watch them get sick.  At Mount Sinai, researchers are conducting a number of studies to learn more about disease and how they can develop innovative treatments to help patients, in areas such as schizophrenia, neurodegeneration disorders and brain cancer.

Areas of Research

Our research in stem cells is focused on a number of different areas.

Modeling Schizophrenia with Stem Cells

Researchers at Mount Sinai have obtained skin cells from patients with schizophrenia and from healthy controls.  Then, they have turned the skin cells into neurons and compared them. Our goal is to observe what is going wrong in neurons from schizophrenia patients, in order to understand why it is happening and discover how to prevent it.  We have learned that in many ways, the brain cells from schizophrenia patients are the same as those from healthy controls.  But in a few important ways, they are different; for example, they don’t connect to each other as well.  Our goal is to use a near limitless supply of live human neurons affected by schizophrenia to screen thousands of drugs, hoping to find one that improves the connections between schizophrenic brain cells.  The hope is that this type of drug might help patients too.

Investigators involved: Kristen Brennand, Pamela Sklar, Schahram AkbarianJoseph Buxbaum, Eric Schadt

Studying Neurodegeneration with Stem Cells

Mutations in critical genes, including APP, PSEN and APOE, substantially increase the risk of Alzheimer’s disease. Likewise, mutations in several genes are known to cause Parkinson’s disease. Mount Sinai researchers are collecting skin samples from patients with these Alzheimer’s or Parkinson’s disease-associated mutations, in order to study, in live human neurons, astrocytes and microglia, exactly how these mutations contribute to neuronal death, and how it can be prevented.

Researchers involved: Samuel E. GandyAlison Goate, Thomas ZwakaTimothy Blenkinsop

Studying the Role of Stem Cells in Brain Cancer

Research teams at Mount Sinai are tackling brain tumors by studying their molecular and cellular components and developing novel strategies to effectively deliver anti-cancer agents to the tumor site. Learn more about brain tumor research at The Friedman Brain Institute.

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