Investigators from the Francis Crick Institute and the UCL Queen Square Institute of Neurology have created DNA molecules which contain special ‘invisibility cloak’ strings to make sure healthy cells cannot read the messages inside.
However, in amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), the invisibility cloak is being lifted and the DNA is reprogram to work its wonders to the cells it affected and hopefully make it better.
This protein is typically found in healthy cells located close to your DNA and is necessary for cells to properly understand the DNA’s genetic blueprint. However in diseased cells the TDP-43 protein translocates to the extreme ends of the cells, far from DNA, which itself alters the ‘reading’ of the messages encoded in genes.
This, the researchers believe, would help make gene therapy treatments safer and more effective since the instructions that help fight disease would only be functional in the minimal amount of sick cells.
This new technology was invented jointly by me and Dr. Anna Williams at the Crick Institute and hopefully we shall be unearthing a much braver therapeutic method for motor neuron disease.
‘If it’s cancer, if it is heart disease or motor neuron disease – the question is always the same; for us, it was what is there about the diseased cells that we can or should exploit – and we chose the TDP-43 protein because it gets messed up in all these neurodegenerative disease states told Dr. Patel.
In addition to its potential for administering gene therapies, our approach can also be employed for identifying cells that are infected or have been transformed in some way.
Which might assist different scientists to check their treatments’ functionality at operating earlier than going straight into a clinical trial.
‘It seems’ affirms Le Pichon, ‘that gene therapy can be of interest for the treatment of neurodegenerative diseases such as ALS and FTD, which occur frequently but for which there are few therapeutic possibilities’. TDP-43 is involved in regulating various aspects of cell viability, and TDP-43 pathological disposal participates prominently in disease development.
Thus, restoring TDP-43 function specifically in the cells which have lost capacity for generating it is a step toward better precision medicine. I think that the more preclinical work is done, the better the chances of successful therapies Consequently, we anticipate further work to replicate and expand on our conclusions.
Reference:
Crick F. DNA molecules with “invisibility cloak” sequences can selectively target diseased cells in motor neuron disease
