Diseases, Conditions, Syndromes

Pushing the boundaries of treatment for Wilson disease

Uyen To, MD, assistant professor of medicine (digestive diseases) and transplant hepatologist, discusses how she first became interested in studying Wilson disease, the wide spectrum of symptoms caused by the rare condition, ...

Genetics

Analytical methods for preclinical stage gene therapy programs

Using a process confirmation vector, researchers developed and optimized a size exclusion chromatography (SEC) with UV and multi-angle light scattering (MALS) method to measure the level of empty capsids during manufacturing. ...

Genetics

Study finds that experts support DNA sequencing in newborns

Babies born in U.S. hospitals currently undergo routine newborn screening, a laboratory test to identify the risk of up to 60 treatable conditions. But hundreds of genetic disorders, including a growing number of devastating ...

Medical research

Scientists deliver siRNA therapy to lung

Scientists at UMass Chan Medical School have developed a technology to deliver gene therapy directly to lung tissue through intranasal administration, a development that could potentially create a new class of treatments ...

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