Dr. Rebecca San Gil from Associate Professor Adam Walker’s lab at UQ’s Queensland Brain Institute has developed a longitudinal map of the proteins involved in MND across the trajectory of the disease, identifying potential therapeutic pathways for further investigation.
“The map is a springboard for many more projects exploring the proteins activated and repressed during the onset, early and late stages of MND,” Dr. San Gil said.
“These proteins are biological factors that drive disease onset and progress its development over time. We measured differences in protein levels in the brain across the trajectory of the disease and collated this information into a longitudinal map.”
The paper is published in Nature Communications. The map is now available for scientists worldwide and will accelerate investigations into MND.
Dr. San Gil has been working in mouse models of MND to understand the mechanisms driving TDP-43 pathology in the brain, which accounts for 95% of amyotrophic lateral sclerosis (ALS) cases and 50% of frontotemporal lobar degeneration (FTLD).
Building on the mapping project, Dr. San Gil chose to focus on a protein-folding factor called DNAJB5.
“Before the onset of MND in mouse models, we observed a marked increase in protein groups responsible for physically assisting in the protein folding process.
“One of these ‘chaperone’ proteins, DNAJB5, was particularly abundant early on, sparking our curiosity about its role in disease progression.
“In human brain tissue, we found DNAJB5 enriched in areas where TDP-43 aggregates.
“The short-term elevation of DNAJB5 is likely a protective mechanism by neurons in an attempt to control TDP-43 as it begins to dysfunction.
“This protective response to TDP-43 needs further investigation because it may help us identify preventative and therapeutic approaches to MND.”
A/Prof. Walker envisions that the lab will continue to follow other identified protein pathways, using gene therapy and repurposing medicine, to see if they can alter or prevent the disease.
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
Skyline Therapeutics Receives FDA Orphan Drug Designation for Gene Therapy for Retinitis Pigmentosa
BOSTON & SHANGHAI, September 2, 2024 – Skyline Therapeutics, a gene therapy company focused on innovative treatments for rare and severe diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SKG1108....
Novavax Receives Emergency Use Authorization for Updated Covid-19 Vaccine
Novavax has received emergency use authorization (EUA) from the U.S. Food and Drug Administration (FDA) for its updated Covid-19 vaccine, NVX-CoV2705, joining other vaccine manufacturers in addressing evolving virus variants. The EUA permits the use of Novavax's...
Turn Biotechnologies Reports Historic Skin Cell Rejuvenation Breakthroughs at ESDR this Week
Company will present data from its successful rejuvenation of fibroblasts and keratinocytes to influential gathering of international dermatologists MOUNTAIN VIEW, Calif., Sept. 4, 2024 /PRNewswire/ -- Turn Biotechnologies, a developer of novel mRNA medicines and...
H5 influenza vaccines: What needs to be done to reduce the risk of a pandemic
As the global threat of H5N1 influenza looms with outbreaks across species and continents including the U.S., three international vaccine and public health experts say it is time to fully resource and support a robust strategy to address this and future potential...
Related Services