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Gene Therapy Weekly

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Amyotrophic Lateral Sclerosis



Findings in amyotrophic lateral sclerosis reported from Mayo Clinic, Medical Department



July 9th, 2009

"Inclusions of TAR DNA-binding protein-43 (TDP-43), a nuclear protein that regulates transcription and RNA splicing, are the defining histopathological feature of frontotemporal lobar degeneration with ubiquitin-positive inclusions (FTLD-Us) and sporadic and familial forms of amyotrophic lateral sclerosis (ALS). In ALS and FTLD-U, aggregated, ubiquitinated, and N-terminally truncated TDP-43 can be isolated from brain tissue rich in neuronal and glial cytoplasmic inclusions," scientists in the United States report.

"The loss of TDP-43 function resulting from inappropriate cleavage, translocation from the nucleus, or its sequestration into inclusions could play important...


Source: Gene Therapy Weekly (2009-07-09)

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