Alzheimer’s Disease Research at NYSCF

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About Alzheimer’s Disease

Alzheimer’s disease is a devastating neurological disease that causes dementia and eventually leads to death. Though symptoms manifest differently in each patient, many early-stage patients become forgetful and confused. As the disease advances, patients can experience long-term memory loss, mood swings, difficulty in speech, and personality changes. 5.4 million people in the United States are living with this disease and 8.2 million more will be diagnosed by 2050.

The two hallmarks of Alzheimer’s pathology are amyloid-beta plaques and neuron tangles—structural abnormalities found in the brain. However, we do not know the exact mechanisms by which these abnormalities lead to the disease, or how big of a role each plays in its onset.

Alzheimer’s Disease Research at NYSCF

At NYSCF, we are studying the actual human cells affected by Alzheimer’s and developing treatment strategies. We are doing this in several ways:

  1. At the NYSCF Research Institute, we are using our own, powerful robotic system for creating stem cells. Our NYSCF Global Stem Cell Array can rapidly, cleanly, and reproducibly create stem cells from skin or blood, and then reprogram them to become the neurons implicated in Alzheimer’s.
  2. Once made, these neurons allow us to investigate the molecular and cellular causes of Alzheimer’s as it develops so that we may see the actual decline and failure of diseased cells.
  3. We can also use these cells to rapidly and efficiently test drug efficacy and potential toxicity, identifying the safest and most promising compounds to advance to clinical trials.

Alzheimer’s Disease Stem Cell News

Publications

Below are select publications outlining recent advancements in Alzheimer’s Disease research from NYSCF Innovators.

CRISPR/Cas9-Correctable mutation-related molecular and physiological phenotypes in iPSC-derived Alzheimer’s PSEN2N141I neurons.
Ortiz-Virumbrales M, Moreno CL, Kruglikov I, Marazuela P, Sproul A, Jacob S, Zimmer M, Paull D, Zhang B, Schadt EE, Ehrlich ME, Tanzi RE, Arancio O, Noggle S, Gandy S.
Acta Neuropathologica Communications. 2017. doi: 10.1186/s40478-017-0475-z.

In this study, NYSCF scientists in collaboration with researchers at the Icahn School of Medicine at Mount Sinai refined a technique to turn skin cells from patients with mild cognitive impairment into the brain cells that degenerate first in Alzheimer’s disease.

Characterization and Molecular Profiling of PSEN1 Familial Alzheimer’s Disease iPSC-Derived Neural Progenitors.
Sproul AA, Jacob S, Pre D, Kim SH, Nestor MW, Navarro-Sobrino M, Santa-Maria I, Zimmer M, Aubry S, Steele JW, Kahler DJ, Dranovsky A, Arancio O, Crary JF, Gandy S, Noggle SA.
PLoS One. 2014. doi: 10.1371/journal.pone.0084547.

This study outlines how NYSCF scientists in collaboration with scientists at the Icahn School of Medicine at Mount Sinai successfully generated a stem cell model of familial Alzheimer’s disease and identified genes that contribute to its pathology and onset.

Generation of iPSC lines from archived non-cryoprotected biobanked dura mater.
Sproul AA, Vensand LB, Dusenberry CR, Jacob S, Vonsattel JP, Paull DJ, Shelanski ML, Crary JF, Noggle SA.
Acta Neuropathologica Communications. 2014. doi: 10.1186/2051-5960-2-4

This paper details how NYSCF scientists were able to generate induced pluripotent stem cell lines from bio-banked tissue taken from Alzheimer’s patients.