How CRISPR is tackling the troubling immune response that’s plagued gene therapy until now

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One of the major challenges facing gene therapy — a way to treat disease by replacing a patient’s defective genes with healthy ones — is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia, and genetic diseases of metabolism. But the technology couldn’t dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

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World-first gene therapy reverses Alzheimer’s memory loss in mice

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Research into Alzheimer’s-related memory loss has uncovered an exciting new breakthrough in the form of a world-first gene therapy

Scientists in Australia have made an exciting breakthrough in Alzheimer’s research, demonstrating what they describe as the first gene-therapy-based approach for treating advanced forms of the disease. Through experiments in mice, the team was able to show how activating a key enzyme in the brain can prevent the kind of memory loss associated with advanced forms of Alzheimer’s, and even reverse it.

The research was carried out at Macquarie University, where dementia researchers and brothers Lars and Arne Ittner were investigating the role of a key enzyme in the brain called p38gamma. Through previous research, the brothers had shown that by activating this enzyme in mice with advanced dementia, they could modify a protein that prevents the development of Alzheimer’s symptoms.

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In surprise result, gene therapy reverses blinding eye disease

The most futuristic predictions that came true in 2017

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The trouble with the future is that it never seems to arrive. That’s why we call it the future. We consequently have this bad habit of taking the present, and all the wondrous and horrific things it has to offer, for granted. As a reminder that we’re actually living in the future of a not-so-distant past, we present to you a list of the most futuristic things that happened in 2017.

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New gene therapy wipes out leukemia in three patients

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The new gene therapy to treat leukemia uses the patients’ own blood cells to hunt down and wipe out their cancer.

The first clear success with gene therapy to treat leukemia, turning the patients’ own blood cells into assasins that hunt down and wipe out their cancer has been reported by scientists at the University of Pennsylvania.

 

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“The Sickle Cell Gene” Supports ‘Malaria Hypothesis”

“Malaria No More” plans on destroying all malaria by 2015!

At a global scale, the sickle cell gene is most commonly found in areas with historically high levels of malaria, adding geographical support to the hypothesis that the gene, while potentially deadly, avoids disappearing through natural selection by providing protection against malaria.

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A New Way to Make Stem Cells and Avoid the Political Pitfalls of Stem Cell Treatments

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A microscope image shows neurons created from pluripotent stem cells using modified RNA.

A Harvard researcher has developed a way to make pluripotent stem cells that solves several of the major impediments to using them to treat human diseases.

 

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Fat and Muscle Tissue Could Be Turned Into Bone and Cartilage

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Muscle cells, pictured, could be encouraged to change into cartilage and then bone by using a form of gene therapy.

Fat and muscle tissue taken from patients’ own bodies could be converted into bone and cartilage to speed up the time it takes to heal injuries.  Researchers have been able to regrow bone and cartilage by inserting a gene into muscle and fat cells and then implanting them at the site of an injury.

Minor Variations in One Gene May Be Associated With Endurance Running

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Elite endurance athletes were more likely to have variations of the NRF2 gene than elite sprinters.

A few minor variations in one gene may make a difference in athletic endurance, according to a new study from Physiological Genomics.

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