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Image: Jessica Bush, a graduate student at the Skaggs Graduate School of Chemical Biology at the Jupiter Research Center in Scripps, Florida, examines her experiments with chemistry professor Dr.
Matthew Disney and associate researcher Dr.
Haruo Aikawa
.
The team published a study in Science Translational Medicine describing a method to target hereditary amyotrophic lateral sclerosis and frontotemporal dementia
Source: Scott Wiseman of Scripps Research
C9 ALS/FTD is one of the most common genetic forms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia
.
It is named after the repeated fragments of DNA on chromosome 9 that cause the disease .
The team’s research was published in the journal Science Translational Medicine on Wednesday
.
Collaborators include Dr.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, causes the progressive loss of motor neurons (the elongated nerve cells that connect muscles to the central nervous system)
.
When a motor neuron dies, it is paralyzed, muscles are lost, swallowing, and eventually, breathing difficulties, which eventually leads to death
Frontotemporal dementia also has familial and sporadic causes
.
It involves progressive damage to neurons in the frontal and temporal lobes of the brain
One disease, multiple symptoms
On the surface, FTD patients and ALS patients have completely different diseases, but those patients caused by C9 gene duplication have the same diseases
.
Performance varies with the type of cells involved
The pathogenic mutation involves the duplication of guanine and cytosine nucleotides, especially the duplication of the GGGGCC fragment on chromosome 9, open reading frame 72
.
The number of repetitions that cause the disease ranges from 60 to thousands
"This is a family-inherited disease
.
Based on the number of repetitions, doctors can assess whether the patient will be infected with the disease
Team effort
To evaluate the effectiveness of these compounds, the team needed both diagnostic biomarkers and patient-derived neurons that showed C9 mutations
.
The Petrucelli team at the Mayo Clinic extensively studied C9 ALS/FTD and developed diagnostic biomarkers
.
Rothstein's research team at Johns Hopkins University treated and studied amyotrophic myelitis and provided stem cells.
The laboratory then implanted these stem cells into neurons showing GGGGCC repeat sequence lesions
.
In their research, the team designed a compound that targets the RNA involved in the transcription of genes that cause disease
.
This compound leads to an interaction between RNA and the way cells use to eliminate RNA
.
This compound eliminates 70% of the disease-causing and toxic protein fragments in mouse bread and removes most of the disease's features from the patient-derived nerve cells
.
Jessica Bush, the first author of this paper and a graduate student at the Skags Institute of Chemical Biology at the Scripps Research Institute, said that one injection in mice, during the entire study period, also In just six weeks, all showed benefits
.
Bush said: "I think this is very exciting.
We can study diseases like ALS.
By adopting different methods or new perspectives, we can open the door to a whole new world full of possibilities.
And began to walk on the road of treatment
.
"
The basic science behind this discovery
The researchers used their nearly 15 years of research results to design this compound
.
The research is to search for drugable structures on RNA (a well-known variable and transient molecule), and establish a compound library that can combine these drugable structures
.
This successful compound binds disease-causing RNA tightly in multiple places while attracting an enzyme to eliminate RNA
.
Obviously, nature created this degrading enzyme to protect cells from virus infection and provide quality control for protein production
.
"We use a natural process to eliminate disease-causing RNA.
A comprehensive analysis of the RNA in cells treated with this compound shows that it is very specific and selective
.
"
To advance this technology to a stage where it can be tested on humans, a large number of additional tests and improvements are needed, and this process may take several years
.
"We hope that these studies will advance new methods that target RNA that causes ALS and other diseases
.
" "We may eventually treat these patients before they develop symptoms, but there is still a long way to go before they reach the clinic.
", has submitted a patent publication application
.
