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Article

Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model.

Neuron - 19 Feb 2020

Nguyen Lien, Montrasio Fabio, Pattamatta Amrutha, Tusi Solaleh Khoramian, Bardhi Olgert, Meyer Kevin D, Hayes Lindsey, Nakamura Katsuya, Banez-Coronel Monica, Coyne Alyssa, Guo Shu, Laboissonniere Lauren A, Gu Yuanzheng, Narayanan Saravanakumar, Smith Benjamin, Nitsch Roger M, Kankel Mark W, Rushe Mia, Rothstein Jeffrey, Zu Tao, Grimm Jan, Ranum Laura P W

Abstract excerpt

The intronic C9orf72 G4C2 expansion, the most common genetic cause of ALS and FTD, produces sense- and antisense-expansion RNAs and six dipeptide repeat-associated, non-ATG (RAN) proteins, but their roles in disease are unclear. We generated high-affinity human antibodies targeting GA or GP RAN proteins. These antibodies cross the blood-brain barrier and co-localize with intracellular RAN aggregates in C9-ALS/FTD...

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Share to Amyotrophic Lateral Sclerosis
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 31831332
DOI: 10.1016/j.neuron.2019.11.007
PMCID: PMC7391607

Genes

RAN
C9
TRIM21

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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