In ALS, the loss of certain proteins turns off a gene called UNC13A that needs to stay active for nerve cell communication, a ...

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that causes progressive muscle weakness. A research ...

Researchers are one step closer to finding a common target for treating ALS - a fatal neurodegenerative disease that causes ...

ALS-associated RNA-binding proteins promote UNC13A transcription through REST downregulation - (https://www.embopress.org/doi ...

UNC13A is an essential regulator of neurotransmitter release at synapses and is one of a number of pre-mRNAs that becomes mis-spliced due to loss of nuclear TDP-43 in disease.

This latest funding will specifically support AcuraStem's continued efforts to advance its targeted therapeutic program for the UNC13A gene, using its iNeuroRx ® technology platform. iNeuroRx ...

QurAlis’ ASOs correct UNC13A mis-splicing, restore UNC13A protein production and reduce cryptic exons that may contribute to disease progression. “We are determined at Lilly to uncover the next great ...

UNC13A is an essential regulator of neurotransmitter release at synapses. In ALS and FTD, TDP-43 accumulates in the cytoplasm and no longer maintains its normal function controlling RNA metabolism ...

Under the terms of the agreement, QurAlis granted Lilly an exclusive, worldwide license to develop and commercialize QRL-204 and other UNC13A-targeting compounds in exchange for an upfront payment ...

AcuraStem's iNeuroRx ® platform has successfully replicated UNC13A pathology in patient neurons, enabling the rapid development and testing of antisense oligonucleotides (ASOs).