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Tag: "faulty" at biology news

Muscle-targeted gene therapy reverses rare muscular dystrophy in mice

...lacement therapy, in which a normal version of the faulty enzyme in those with the disease is infused weekly. In clinical trials of the replacement therapy, the infusions have already prolonged the lives of many babies with Pompe disease, Koeberl said. "A number of babies have been receiving enzyme replace...

Circles Of DNA Might Help Predict Success Of Stem Cell Transplantation

...n stem cells that produce cancerous white cells or faulty red cells and replace them with healthy stem cells from donors. If the transplants succeed, the donated stem cells repopulate the blood with healthy red and white cells. The St. Jude team showed that the more copies of tiny rings of DNA called signa...

Silence the gene, save the cell: RNA interference as promising therapy for ALS

...l identifies the resulting messenger RNA strand as faulty and chops it up. As a result, the genetic blueprint isn't delivered and the protein never gets made. "Gene silencing is an example of using "molecular scissors" at its most advanced level," Raoul explains. Raoul and colleagues used RNA interference...

Cerebral navigation: How do nerve fibers know what direction to grow in?

...ns grew into the hind limb prematurely, indicating faulty axon guidance. "Understanding these pathways could help in understanding the process of nerve regeneration," says Greenberg, who is also Professor of Neurology and Neurobiology at Harvard Medical School. "The mechanisms we've uncovered could provide...

Genetic therapy reverses nervous system damage in animal model of inherited human disease

...omes. In the case of AMD, children are born with a faulty version of the gene for an enzyme called lysosomal alpha-mannosidase or LAMAN. "Shortly after birth, brain tissue is still physically maturing, which means that there is a particularly important window of opportunity for gene therapy in infants," Wo...

Hopkins scientists uncover 'tags' that force proteins to cell surface

...t detect odors in the nose, and the protein that's faulty in cystic fibrosis. Being able to force these to t...s Li. "But now we know the problem might just be a faulty transportation signal." Given that proteins can be thousands of building blocks long, the final eig...

Once given 'no respect,' cells' tiny RNAS take driver's seat

... which they are used to make proteins. Myc also is faulty and overactive in many human cancer cells, althoug... cancer in mice predisposed to cancer because of a faulty Myc gene. "We've found that there's complex crosstalk between Myc, the microRNAs and the genes that...

Huntington's cure in flies lays groundwork for broader treatment approaches

... which are set in motion, for example, by a single faulty gene. Yin has long worked in the area of "triplet expansion" diseases such as Huntington's and Fragile X, in which genes go haywire due to a coding defect. His collaborators on the recent fruit fly work include, among others, lead author Kanae Iijim...

New study to explore cellular circuitry

...e re-wired so that existing components replace the faulty circuit. In addition, researchers now believe that modules with related functions are grouped together in networks. "With most genes redundant and related to other genes and proteins in predictable ways, we can begin to identify the function of unk...

Dendritic cells offer new therapeutic target for drugs to treat MS and other autoimmune disease

...elieve that targeting dendritic cells may stop the faulty immune response at a higher "upstream" level since T-cells frequently receive their information from dendritic cells. Testing their idea, Small and his Hopkins colleague Katherine Whartenby used an experimental compound called CEP-701, already know...

Study provides insight into cellular defenses against genetic mutation

...s," Maquat said. "Beyond its role in screening out faulty mRNAs through immediate decay, we have also learned that the NMD factor Upf1 is part of a related process that regulates the expression of many normal genes by controlling the timeframe in which their encoded mRNAs decay. Evolution often borrows a us...

Scientists pinpoint inflammation gene

..., Blangero said. Inflammation develops when those faulty proteins accumulate in a cell. People with a gene...oping an understanding of why obese persons with a faulty SEPS1 gene may be at higher risk of developing heart disease and diabetes," he said. The path to d...

New battery technology helps stimulate nerves

...e injected near target nerves to help overcome the faulty nervous system wiring at the heart of Parkinson's, epilepsy and incontinence. "The idea is that whenever you have a broken nerve connection, you can supply the electrical impulse to complete the circuit," West explains. The microstimulator was deve...

Ancient DNA helps UF researchers unearth potential hemophilia therapy

...or millions of years, the researchers replaced the faulty gene responsible for the disease in neonatal mice, according to findings published online this month in the journal Molecular Therapy. "The degree to which these patients have problems from hemophilia stems from how much of this protein, factor VIII...

Cloned stem cells prove identical to fertilized stem cells

...m cells in the blastocyst appear to be spared this faulty reprogramming. When stem cells from a cloned blastocyst are removed and placed into a dish, most die. A few, however, survive and give rise to an embryonic stem cell line, and these appear to be thoroughly reprogrammed. Researchers have tried to te...

Common enzyme is a key player in DNA repair

...ocesses, which can surgically excise and replace a faulty section. But sometimes a damaged bit of DNA slips through unrepaired. A faulty nucleotide--the basic constituent of DNA--can stall the temperamental DNA replication machinery as i...

Nobelist discovers antidepressant protein in mouse brain

...d similar psychiatric illnesses thought to involve faulty serotonin receptors. To test this idea, the researchers examined p11 levels in the brains of depressed humans and "helpless" mice, considered a model of depression since they exhibit behaviors similar to those of depressed humans. They compared thes...

Faults in newly discovered breast stem cells may lead to tumours

...e breast stem cell or a "daughter" cell to produce faulty cells. In effect, the errant breast cell can becom...will be designed to engage with and neutralize the faulty feature of the stem cell. Further research is now under way on excised human breast tumours to con...

Efficiency, not more doctors, is the prescription for aging population

...or expanding the physician workforce is based on a faulty assumption, according to the authors. Proponents reason that the practices of the highest-intensity medical centers where many more doctors and resources are used in providing medical services at the end of life, should be the standard for the count...

UF scientists test improved gene therapy method for hereditary heart conditions

...e treatment of cardiovascular conditions linked to faulty genes or congenital metabolic diseases, including atherosclerosis, stroke, muscular dystrophy and an enlargement of the heart muscle known as dilated cardiomyopathy. But efforts to begin testing it in people have been slowed by the need to find way...

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