Survival and Motor Phenotypes in FVB C9-500 ALS/FTD BAC Transgenic Mice Reproduced by Multiple Labs.

Nguyen, Lien; Laboissonniere, Lauren A; Guo, Shu; Pilotto, Federica; Scheidegger, Olivier; Oestmann, Angelina; Hammond, Jennetta W; Li, Herman; Hyysalo, Anu; Peltola, Roosa; Pattamatta, Amrutha; Zu, Tao; Voutilainen, Merja H; Gelbard, Harris A; Saxena, Smita; Ranum, Laura P W (2020). Survival and Motor Phenotypes in FVB C9-500 ALS/FTD BAC Transgenic Mice Reproduced by Multiple Labs. Neuron, 108(4), 784-796.e3. Cell Press 10.1016/j.neuron.2020.09.009

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Mordes et al. (2020) did not detect the survival or motor phenotypes in C9orf72 BAC transgenic mice originally described by Liu et al. (2016). We discuss methodological differences between the Mordes and Liu studies, several additional studies in which survival and motor phenotypes were found, and possible environmental and genetic effects. First, Nguyen et al. (2020) showed robust ALS/FTD phenotypes in C9-BAC versus non-transgenic (NT) mice and that α-GA1 treatment improved survival, behavior, and neurodegeneration. The groups of Gelbard and Saxena also show decreased survival of C9-BAC versus NT mice and neuropathological and behavioral deficits similar to those shown by Liu et al. (2016). Although FVB/N mice can have seizures, increases in seizure severity and death of C9 and NT animals, which may mask C9 disease phenotypes, have been observed in recent C9-500 FVB/NJ-bred cohorts. In summary, we provide an update on phenotypes seen in FVB C9-BAC mice and additional details to successfully use this model. This Matters Arising Response paper addresses the Mordes et al. (2020) Matters Arising paper, published concurrently in Neuron.

Item Type:

Journal Article (Original Article)


04 Faculty of Medicine > Department of Head Organs and Neurology (DKNS) > Clinic of Neurology

Graduate School:

Graduate School for Cellular and Biomedical Sciences (GCB)

UniBE Contributor:

Pilotto, Federica, Scheidegger, Olivier, Oestmann, Angelina, Saxena, Smita


600 Technology > 610 Medicine & health




Cell Press




Chantal Kottler

Date Deposited:

06 Nov 2020 16:46

Last Modified:

05 Dec 2022 15:41

Publisher DOI:


PubMed ID:


Uncontrolled Keywords:

ALS/FTD C9orf72 FVB strain background RAN proteins microsatellite repeat motor neuron disease repeat associated non-AUG (RAN) translation repeat expansion mutation seizures transgenic mouse models




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