Journal
NEURON
Volume 108, Issue 5, Pages 822-842Publisher
CELL PRESS
DOI: 10.1016/j.neuron.2020.08.022
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Funding
- NIH [R35NS097263, R01AG064690]
- Robert Packard Center for ALS Research at Johns Hopkins
- Brain Rejuvenation Project of the Wu Tsai Neurosciences Institute
- Stanford Knight-Hennessy Scholars Program
- National Science Foundation (NSF) Graduate Research Fellowship [DGE-1656518]
- Stanford Graduate Fellowship
- Stanford Genome Training Program [5T32HG000044-23]
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Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder caused by the loss of motor neurons from the brain and spinal cord. The ALS community has made remarkable strides over three decades by identifying novel familial mutations, generating animal models, elucidating molecular mechanisms, and ultimately developing promising new therapeutic approaches. Some of these approaches reduce the expression of mutant genes and are in human clinical trials, highlighting the need to carefully consider the normal functions of these genes and potential contribution of gene loss-of-function to ALS. Here, we highlight known loss-of-function mechanisms underlying ALS, potential consequences of lowering levels of gene products, and the need to consider both gain and loss of function to develop safe and effective therapeutic strategies.
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