The Maragakis lab's main focus is in understanding disease mechanisms and targeting cell therapeutics for Amyotrophic Lateral Sclerosis (ALS). In collaboration with Johns Hopkins neuroscientists, his laboratory helped create stem cell lines from ALS patients using induced pluripotent stem cell (iPSC) methodologies. These cells will allow for the development of human cell lines which can be used for both the basic understanding of ALS astrocyte and motor neuron biology, as well as eventually identifying ALS therapeutics. Researchers in the Maragakis lab also focus on the potential therapeutic role of astrocyte replacement in ALS using glial stem cells. By transplanting glial stem cells into ALS animal models, the researchers in the Maragakis lab found that the stem cells can engraft, migrate and differentiate into astrocytes, and subsequently provide neuroprotection to vulnerable motor neuron pools. The team uses stem cell transplantation biology to understand the influences of mutant SOD1 astrocytes on normal, healthy motor neurons. A significant laboratory effort is underway to translate these discoveries into therapies for patients with ALS.
Lab Website: ALS Center
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Taga A, Dastgheyb R, Habela C, Joseph J, Richard J-P, Gross SK, Lauria G, Lee G, Haughey N, Maragakis NJ. Role of human induced pluripotent stem cell-derived spinal cord astrocytes in the functional maturation of motor neurons in a multielectrode array system. Stem Cells Transl Med. 2019 Dec;8(12):1272-1285. doi: 10.1002/sctm.19-0147. Epub 2019 Oct 21 PMID:31631575 PMCID:PMC6877769
Almad AA, Doreswamy A, Gross SK, Richard JP, Huo Y, Haughey N, Maragakis NJ. Glia. 2016 Apr 16. doi: 10.1002/glia.22989. Connexin 43 in astrocytes contributes to motor neuron toxicity in amyotrophic lateral sclerosis.
Haidet-Phillips AM, Doreswamy A, Gross SK, Tang X, Campanelli JT, Maragakis NJ. Human glial progenitor engraftment and gene expression is independent of the ALS environment. Exp Neurol. 2015 Feb;264:188-99. PubMed PMID: 25523812.
Haidet-Phillips AM, Roybon L, Gross SK, Tuteja A, Donnelly CJ, Richard JP, Ko M, Sherman A, Eggan K, Henderson CE, Maragakis NJ. Gene profiling of human induced pluripotent stem cell-derived astrocyte progenitors following spinal cord engraftment. Stem Cells Transl Med. 2014 May;3(5):575-85. PubMed PMID: 24604284; PubMed Central PMCID: PMC4006486.
5.Haidet-Phillips AM, Gross SK, Williams T, Tuteja A, Sherman A, Ko M, Jeong YH, Wong PC, Maragakis NJ. Altered astrocytic expression of TDP-43 does not influence motor neuron survival. Exp Neurol. 2013 Dec;250:250-9. PubMed PMID: 24120466.