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

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 ...

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.

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

UNC13A is also one of the mRNA sequences that contained cryptic exons when TDP-43 was reduced, and cryptic exons were also seen in neurons taken from postmortem tissue of ALS and FTD patients.

About UNC13A UNC13A is an essential regulator of neurotransmitter release at synapses. In ALS and FTD, the loss of TDP-43 causes the mis-splicing of certain pre-mRNA transcripts resulting in ...

Acurastem Inc. has secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to facilitate the development of its UNC13A program toward clinical trials for ...

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 ...