New Research Finds That Designer DNA Drugs May Help Delay Paralysis Among ALS Patients

And when there is reduced TDP-43 activity, there is a misassembly of stathmin-2– or a protein necessary for the maintenance of connections between motor neurons and muscle.

If there is no stathmin-2, motor neurons ultimately disconnect from the muscle and lead to paralysis– which is characteristic of ALS. What Cleveland and his team found, though, was that they could use a designer DNA drug to mimic the TDP-43 function. This then restores correct stathmin-2 RNA and protein levels in the nervous system.

To do this, the researchers edited mice genes to contain STMN2 gene sequences. Then, they injected oligonucleotides into the cerebral spinal fluid. Oligonucleotides are small pieces of DNA or RNA that are able to bind to distinct RNA molecules. This blocks the RNA molecules’ ability to make a protein or to change how final RNAs are assembled.

The injections were able to correct STMN2 pre-mRNA misprocessing while restoring stathmin-2 protein expression. And remarkably, this was accomplished completely independently of the TDP-43 function.

Now, Cleveland believes his team’s findings could have significant implications for delaying paralysis among ALS patients.

“Our findings lay the foundation for a clinical trial to delay paralysis in ALS by maintaining stathmin-2 protein levels in patients using our designer DNA drug,” he concluded.

To read the study’s complete findings, which have since been published in Science, visit the link here.

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