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Gabsang Lee & Nicholas Maragakis

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Gabsang Lee, PhD, DVM & Nicholas Maragakis, MD

Johns Hopkins UniversityJohns Hopkins University

Derivation of oculomotor neurons using PHOX2b::GPF reporter ALS-hiPSC lines

In ALS patients in the most advanced stages of illness, nearly all of their motor neurons are affected, except the ones that are responsible to move their eyes. Understanding why the motor neurons in the eyes remain functional when spinal motor neurons degenerate and die could provide valuable information on ALS. Here we will use patient iPSCs to generate ALS-specific eye-motor neurons, which can provide valuable information for understanding ALS pathogenesis and identifying a potential therapeutic target.

 

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Steven Finkbeiner

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Gladstone Institutes, UCSF
Two recently discovered genes that have been associated with both familial and sporadic forms of ALS encode the related proteins TDP43 and FUS cause neuron death in ALS.
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