期刊
NATURE BIOTECHNOLOGY
卷 29, 期 9, 页码 824-U79出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nbt.1957
关键词
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资金
- US National Institutes of Health (NIH) [R01 NS644912-1A1, RC2 NS69476-01]
- Project A.L.S.
- Packard Center for ALS Research [P2ALS]
- Helping Link Foundation
- NIH [NRSAF31NS058224]
- Swiss National Science Foundation
Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease, with astrocytes implicated as contributing substantially to motor neuron death in familial (F)ALS(1-5). However, the proposed role of astrocytes in the pathology of ALS derives in part from rodent models of FALS based upon dominant mutations within the superoxide dismutase 1 (SOD1) gene, which account for <2% of all ALS cases(2,4,5). Their role in sporadic (S)ALS, which affects >90% of ALS patients, remains to be established. Using astrocytes generated from postmortem tissue from both FALS and SALS patients, we show that astrocytes derived from both patient groups are similarly toxic to motor neurons. We also demonstrate that SOD1 is a viable target for SALS, as its knockdown significantly attenuates astrocyte-mediated toxicity toward motor neurons. Our data highlight astrocytes as a non-cell autonomous component in SALS and provide an in vitro model system to investigate common disease mechanisms and evaluate potential therapies for SALS and FALS.
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