New Therapeutic Approach may Make Lou Gehrig's Disease History

A novel therapeutic approach has been identified by scientists for the most frequent genetic cause of ALS.

ALS is a disorder of the regions of the brain and spinal cord that control voluntary muscle movement, and frontal temporal degeneration.

Scientists Explore Link Between Early Balding and Lou Gehrig's Disease
Early balding may be associated with an increased risk of the rare but incurable disease amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig's disease, says study.

The study establishes using segments of genetic material called antisense oligonucleotides - ASOs - to block the buildup and selectively degrade the toxic RNA that contributes to the most common form of ALS, without affecting the normal RNA produced from the same gene.

The new approach may also have the potential to treat frontotemporal degeneration or frontotemporal dementia (FTD), a brain disorder characterized by changes in behavior and personality, language and motor skills that also causes degeneration of regions of the brain.

First author Clotilde Lagier-Tourenne, MD, PhD, UC San Diego Department of Neurosciences and Ludwig Institute for Cancer Research, said that remarkably they found two distinct sets of RNA foci, one containing RNAs transcribed in the sense direction and the other containing anti-sense RNAs.

Genetic Suspects in Sporadic Lou Gehrig's Disease Identified
Mutations in several new genes that might be associated with the development of amyotrophic lateral sclerosis, or ALS have been discovered by scientists.

The researchers also discovered a signature of changes in expression of other genes that accompanies expression of the repeat-containing RNAs.

Since they found that reducing the level of expression of the C9orf72 gene in a normal adult nervous system did not produce this signature of changes, the evidence demonstrated a toxicity of the repeat-containing RNAs that could be relieved by reducing the levels of those toxic RNAs.

Novel Stem Cell Approach Holds Promise in Developing Treatments for Lou Gehrig's Disease
A novel stem cell based approach could be effective in developing treatments for Lou Gehrig's disease, a new study published in the journal Cell Stem Cell reveals.

Lagier-Tourenne said that this led to their use of the ASOs to target the sense strand. We reduced the accumulation of expanded RNA foci and corrected the sense strand of the gene.

She said that importantly, they showed that they could remove the toxic RNA without affecting the normal RNA that encodes the C9orf72 protein.

Researchers Identify New Type of Central Nervous System Cell Critical for Development of Lou Gehrig's Disease
Researchers have identified a type of central nervous system cell, other than motor neurons, which plays an important role in the development of a fatal degenerative disease.

Lagier-Tourenne added that this selective silencing of a toxic RNA is the holy grail of gene silencing approaches, and they showed that they had accomplished it.

The study has been published online in the journal PNAS.

Source-ANI