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World’s First Gene Therapy Renders Impressive Results

Alzheimer’s remains to be one of the more disastrous diseases amongst the wide variety of diseases that are known to man. Due to the very nature of the disease, the psychological impact that it has been known to make is not only limited to the patient themselves, but also to others that surrounded by the person in question. But, what exactly is Alzheimer’s, and what does it do? 

Well, Alzheimer’s is basically a progressive disorder which has the capabilities to make the brain cells go to waste and so – degenerate. The disease remains to be amongst the more common causes of dementia, which happens to be the continuous decline in thinking behavioral as well as social skills. 

Scientists that base themselves in Australia have gone on to make a very exciting breakthrough in the extensive research reflecting on Alzheimer’s. The research goes on to conclude what they describe as the first gene-therapy-based approach for the sake of treating the disease. As the experiments were carried out on mice, it was shown that via the activation of a key enzyme found in the brain, the memory loss which has been associated with Alzheimer’s can affectively be prevented – and even reversed.

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The research was in fact carried out at Macquarie University, where researchers that base themselves around the research of dementia together with two brothers in Lars and Arne Ittner were keen to investigate the role that a key enzyme going by the name of p38gamma plays within the brain. Previous research which was carried out by the brothers went on to showcase that by actually activating this particular enzyme in mice with advanced type of dementia, they could actually proceed on to modify a protein which has the capabilities to prevent the development of Alzheimer’s symptoms.

As the scientists were determined to build upon all the data that they had gathered up with regards to what the experimentation on mice showed off, they were looking to see just how cognitive decline could not only be slowed, but also so as to how the function of this particular enzyme might be restored to levels which are deemed to be normal for the sake of even greater benefit.

Lars Ittner, one of the above mentioned brothers, said : “The naturally protective enzymatic activity in the brain is unfortunately lost the further you progress down the Alzheimer’s disease track – so the more memory you lose, the more you also lose this natural protective effect.”

With the introduction of genetic material, the researcher also made the discovery that not only could they actually activate the p38gamma enzyme in way thereby which memory decline could be stopped, but also go on to actively improve their memory despite the advanced nature that the disease they have contracted poses.

Lars continued by saying : “We were completely surprised. They actually recovered their memory function and their ability to learn returned. So, two months after we treated the mice at very old ages, these mice suddenly behaved like their normal siblings. There is no comparable therapy out there and no other gene therapy either.”

Of course all the discoveries as well as the data which has been collected via this whole does in fact bring about an element of excitement, there is still a lot at stake before such developments may be brought forward in the world of clinical use. However though, it is also important to note that work is already underway thereto determine the very best pathway which may lead towards clinical trials as the team would eye commercialization soon after, perhaps five to ten years down the lane. With that said, more excitement is associated with these latest events due to the fact that the technique’s potential might not just be limited to Alzheimer’s, as the team which has collected all the data also remain hopeful that it could actually end up treating other forms of dementia-related diseases : the prime example of which comes in the form of fronto-temporal dementia – which typically affects younger people aged between that of 40 and 65.

Furthermore, Arne Ittner said : “The brain is a black box and some days we get lucky and get glimpses of how it functions and we learn we can interfere with the mechanism in this black box. Now we have detailed understanding of the mechanisms involved down to amino acids, which is quite unprecedented.”

The dangers associated with Alzheimer’s as well as dementia as whole have been well documented, and scientists have ever since spent an exceptional amount of time in picking out solutions to either eradicate or at least mitigate the problems that associate themselves with the disease. And so as a result, one would have imagine that it is natural that an array of excitement is brought on due to the progressive nature of research that has been carried out with regards to the whole case – as we only hope that there is light at the end of the tunnel for all the patients as well as their families and loved ones who have been ruined as a result of the disease and all the risks that it poses.


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