Medical research

An 'unconventional' path to correcting cystic fibrosis

Researchers have identified an unconventional path that may correct the defect underlying cystic fibrosis, according to a report in the September 2nd issue of the journal Cell. This new treatment dramatically extends the ...

Medical research

Molecular delivery truck serves gene therapy cocktail

In a kind of molecular gymnastics, scientists at the University of North Carolina at Chapel Hill School of Medicine have devised a gene therapy cocktail that has the potential to treat some inherited diseases associated with ...

Neuroscience

Scientists zero in on treatment for Charcot-Marie-Tooth disease

About 1 in 2,500 people have a degenerative nerve disease called Charcot-Marie-Tooth (CMT). The disease is typically diagnosed in children, who can lose their ability to walk and use their hands for fine motor skills. There ...

Diseases, Conditions, Syndromes

Promising treatment for progeria within reach

Pharmaceuticals that inhibit a specific enzyme may be useful in treating progeria, or accelerated aging in children. A new study performed at the Sahlgrenska Academy indicates that the development of progeria in mice was ...

Medical research

Exercise rescues mutated neural stem cells

CHARGE syndrome is a severe developmental disorder affecting multiple organs. It affects 1 in 8500 newborns worldwide. The majority of patients carry a mutation in a gene called CHD7. How this single mutation leads to the ...

Neuroscience

New mouse model of ALS more closely mimics human disease

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a progressive, fatal neurological disease that attacks the nerve cells controlling voluntary muscles. No effective treatments have been found.

Genetics

Scientists tackle Huntington's disease by targeting mutant gene

Huntington's disease is an inherited, neurodegenerative disorder that usually appears in mid-adult life and leads to uncoordinated body movements and cognitive decline. The disease is due to multiple repetitions of a deoxyribonucleic ...

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