Month: <span>March 2020</span>

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Viruses for the Good: Gene Therapy for Sickle Cell Disease
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Viruses for the Good: Gene Therapy for Sickle Cell Disease

While the world worries about novel coronavirus SARS-CoV-2, other viruses continue to be used for the good – as vectors that ferry in healing genes for gene therapy and editing. Charles Hough calls himself “reborn” after lentiviruses – disabled versions of HIV – gave his blood cells the gene that overrides the mutant one that...

Exploring cells’ path to build cholesterol and finding a future drug target
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Exploring cells’ path to build cholesterol and finding a future drug target

Researchers based at UTokyo and RIKEN in Japan, and the University of New South Wales in Australia have uncovered a new aspect of one of the molecules involved in cells’ production line to build cholesterol. This understanding could provide a new target for high-cholesterol medications and other drugs that kill pests like athlete’s foot fungus...

Could disease pathogens be the dark matter behind Alzheimer’s disease?
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Could disease pathogens be the dark matter behind Alzheimer’s disease?

by Richard Harth, Arizona State University For researchers investigating Alzheimer’s Disease (AD), a devastating neurodegenerative illness afflicting close to 6 million Americans, it is the best and worst of times. Scientists have made exponential advances in understanding many aspects of the mysterious disease since it was first diagnosed over 100 years ago. Nevertheless, every effort...

How gene therapy may hold key to treating life-threatening cardiac disease
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How gene therapy may hold key to treating life-threatening cardiac disease

by University of California – San Diego Danon disease is a very rare, life-threatening condition where the fundamental biological process of removing and recycling proteins does not work. This impairment results in dysfunction of the heart, skeletal muscle, neurologic system, eyes, and liver. Most patients die or require heart transplants by the third decade of...

In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1
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In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1

Findings from a phase 2 clinical trial show that the drug selumetinib improves outcomes for children with the genetic disorder neurofibromatosis type 1 (NF1). In the trial, selumetinib shrank the inoperable tumors that develop with NF1 called plexiform neurofibromas, and children experienced reduced pain, improved function, and better overall quality of life after receiving the...

How to slow down ageing?
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How to slow down ageing?

ESTONIAN RESEARCH COUNCIL Healthy ageing has become one of the priorities of research in Europe. University of Tartu researchers looked for differences in the immune systems of young and old people. They focused on monocytes and found that the monocytes of the elderly do not seem to produce as much energy, and there is an...