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New era in Gene Therapy opens; Link between Pak-4 protein & Parkinson's confirmed

 
The mapping and sequencing of the human DNA has been pointed out as the key to finding effective treatment methods for incurable or degenerative diseases.
 
For IT and Science Front this week, our science correspondent Kim Ji-yeon meet the brightest minds in Korea making great strides in opening a whole new era in gene therapy.
 
The potential of gene therapy, or the therapeutic delivery of drugs into a patient's cells to treat disease, is being revisited as new advances are being made in the field.
 
A study by a team of researchers at Chungbuk National University says it has confirmed a link between a specific type of protein called PAK-4 and Parkinson's disease, which leads to motor dysfunction, tremors and stiffness... as a result of damage to more than 50-percent of brain neurons.
 
The study is ultimately aimed at finding an effective treatment for the disease, which like every other degenerative disease currently has no fundamental cure, and treatment methods are narrowed down to halting or slowing down its symptoms.
The study focuses on the role of a specific type of protein and its relationship to Parkinson's disease."
 
The team found that in early experiments with rats, the PAK-4 protein not only helped mitigate the symptoms of the disease, but also showed the potential to prevent the illness.
 
link between pak4 protein and parkinsons confirmed
 
Since proteins easily disintegrate once inside the human body,... gene therapy could be used to deliver the protein to the cells of Parkinson's disease patients.
 
"Since the experiment used small rodents... more comprehensive research needs to be done on mid-sized animals... such as monkeys since they have a lot of similarities with humans. And if the protein still proves to be effective, it needs to be tested in clinical trials before the gene therapy could be commercialized. All in all, it could take at least ten years of research before it's actually used to treat Parkinson's disease patients."
 
Despite the promising outlook of gene therapy in curing degenerative diseases, it could be useless without an accurate database of human genome or map of the human DNA.
 
To address this obstacle, another team of researchers at the Ulsan National Institute of Science and Technology unveiled the KOREF, a genomic database shared among Koreans... by combining genome data of 41 people.
 
The team says it has successfully narrowed the number of variants among ethnic Koreans from the previous 4-million to 3-million variants when compared to the KOREF.
 
"Since the map includes genome data of Koreans, it would be more useful than just using the standard and currently widespread Caucasian based genome data in diagnosing genetic diseases or developing new treatment methods."
The KOREF is also the first of its kind representing a specific population.
 
The team says it plans to expand the number to at least 10-thousand samples to boost its accuracy and representativeness.
 

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