Function of UCHL1 Gene in Neurons using TALEN-mediated Mutagenesis

Date
2014-04-15
Authors
Horton, Mary
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Abstract

Amyotrophic lateral sclerosis (or Lou Gehrig's disease) is a neurodegenerative disease caused by the death of motor neurons in the spinal cord and the brain. Once these nerve cells die, the patient's muscle cells degenerate, resulting in paralysis and eventually death. Most cases of ALS are sporadic, meaning that a clear molecular and genetic understanding of the mechanisms by which the motor neurons die is lacking. Ubiquitin C-terminal hydrolase-L1 (UCHL1) is one gene that has been implicated in ALS, as well as several other neurodegenerative diseases. To gain a better understanding into the function of UCHL1 in non-diseased neurons, we are using TAL-effector nucleases (TALEN) to create UCHL1 mutant zebrafish. Specifically, I have generated these TALENs and begun to inject them to generate the mutant fish. Once created, these fish will provide insights into the normal function of UCHL1 and, hopefully, allow us to create a new zebrafish model of nerve cell degeneration.

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