BioCentury | Jun 7, 2018
Translation in Brief

New Therapeutic Targets and Biomarkers: May 2018

...fusion (involved in t(12;16) in malignant liposarcoma) (FUS; TLS) mutations and multisystem proteinopathy caused by heterogeneous nuclear ribonucleoprotein A1 (HNRNPA1)...
BioCentury | May 23, 2018
Distillery Therapeutics

Neurology; musculoskeletal

...of TNPO1 could help treat ALS caused by FUS mutations and multisystem proteinopathy caused by HNRNPA1...
...In vitro , TNPO1 decreased the formation of new toxic fibrils from mutant FUS or HNRNPA1...
...survival compared with no transgene. In a Drosophila model of multisystem proteinopathy expressing mutant human HNRNPA1...
BioCentury | Nov 21, 2013
Distillery Therapeutics

Indication: Neurology

...from ALS patient fibroblasts carrying the repeats, RNA foci formed and sequestered RNA-binding proteins, including heterogeneous nuclear ribonucleoprotein A1 (HNRNPA1)...
BioCentury | Nov 7, 2013
Cover Story

ALS antisense oligonucleotides

...including RNA foci formation in the neurons. The foci often colocalized with RNA-binding proteins including heterogeneous nuclear ribonucleoprotein A1 (HNRNPA1)...
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