Tag Archives: mouse model

Gene therapy leads to robust improvements in animal model of fatal muscle disease

Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital childhood disease know as X-linked myotubular myopathy.

The findings, appearing  as the cover story in the January 22, 2014 issue of Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for this devastating disease. (more…)

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Multiple Sclerosis Study Reveals How Killer T Cells Learn to Recognize Nerve Fiber Insulators

Misguided killer T cells may be the missing link in sustained tissue damage in the brains and spines of people with multiple sclerosis, findings from the University of Washington reveal. Cytoxic T cells, also known as CD8+ T cells, are white blood cells that normally are in the body’s arsenal to fight disease.

Multiple sclerosis is characterized by inflamed lesions that damage the insulation surrounding nerve fibers and destroy the axons, electrical impulse conductors that look like long, branching projections. Affected nerves fail to transmit signals effectively. (more…)

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Promising Nanotechnology

Nanotechnology for drug delivery shows promise in treatment of pediatric leukemia

Nanotechnology developed by Delaware scientists could potentially deliver chemotherapy to children in a way that attacks cancer cells without harming healthy cells, greatly reducing side effects.

The work, conducted by researchers in the University of Delaware’s Department of Materials Science and Engineering and Nemours Center for Childhood Cancer Research, was published this month in the journal Molecular Pharmaceutics. (more…)

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Researchers Report Potential New Treatment to Stop Alzheimer’s Disease

Molecular ‘tweezers’ break up toxic aggregations of proteins in mouse model

Last March, researchers at UCLA reported the development of a molecular compound called CLR01 that prevented toxic proteins associated with Parkinson’s disease from binding together and killing the brain’s neurons.

Building on those findings, they have now turned their attention to Alzheimer’s disease, which is thought to be caused by a similar toxic aggregation or clumping, but with different proteins, especially amyloid-beta and tau. (more…)

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Yale Study: How Mitochondrial DNA Defects Cause Inherited Deafness

Yale scientists have discovered the molecular pathway by which maternally inherited deafness appears to occur: Mitochondrial DNA mutations trigger a signaling cascade, resulting in programmed cell death. The study is in the Feb. 17 issue of Cell.

Mitochondria are cellular structures that function as “cellular power plants” because they generate most of the cell’s supply of energy. They contain DNA inherited from one’s mother. Mitochondria determine whether a cell lives or dies via the process of programmed cell death, or apoptosis. (more…)

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