New genetic link found for some forms of SIDS

Some forms of sudden infant death syndrome stem from a genetic mutation that keeps infants from processing lipids in milk, a new study has discovered. The build-up of unprocessed fatty material disrupts heart functions. While no treatments are yet available, the finding could help in genetic screening. Drugs are also being tested to see if they can help.

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Fruit flies help in the development of personalized medicine

It is common knowledge that there is a connection between our genes and the risk of developing certain diseases. In a study on fruit flies, researchers have found that gene mapping can also be used to predict response to a given treatment. This knowledge is crucial for the development of personalized medicine.

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New neurodevelopmental syndrome and NKAP gene

Researchers have identified a gene mutation that causes developmental delay, intellectual disability, behavioral abnormalities and musculoskeletal problems in children. The newly diagnosed condition, called NKAP-related syndrome, arises from mutations in the NKAP gene, which plays a key role in human development.

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Laser therapy gains credibility as effective option for treating vaginal problems

Nearly 50% of menopausal women complain of vaginal dryness, itching, and burning, among other commonly reported menopause symptoms. Laser therapy is one of the newer techniques for addressing these problems. A new study suggests that it is as effective and safe as vaginal estrogen in improving sexual and urinary functionality.

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Researchers create new viral vector for improved gene therapy in sickle cell disease

Researchers have developed a new and improved viral vector — a virus-based vehicle that delivers therapeutic genes — for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times more efficient at incorporating corrective genes into bone marrow stem cells than the conventional vectors currently used, and it had a carrying capacity of up to six times higher, the researchers report.

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Researchers advance search for safer, easier way to deliver vision-saving gene therapy

In experiments with rats, pigs and monkeys, researchers have developed a way to deliver sight-saving gene therapy to the retina. If proved safe and effective in humans, the technique could provide a new, more permanent therapeutic option for patients with common diseases such as wet age-related macular degeneration (AMD), and it could potentially replace defective genes in patients with inherited retinal disease.

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Researchers developing new 'DNA stitch' to treat muscular dystrophy

A new therapeutic being tested is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD). The treatment — a cocktail of DNA-like molecules — results in dramatic regrowth of a protein called dystrophin, which acts as a support beam to keep muscles strong. The protein is virtually absent in those with DMD.

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