Researchers at Nationwide Children’s Hospital report a gene therapy strategy that improves the condition of a mouse model of an inherited blood disorder, Beta Thalassemia. The gene correction involves using unfertilized eggs from afflicted mice to produce a batch of embryonic stem cell lines.
A study appearing in Human Molecular Genetics is one of the first to suggest that low maternal cholesterol could have an impact on pregnancy, possibly reducing the size of the placenta.
A hormone most commonly known for its role in aiding digestion may also help protect the developing brain from the negative effects of alcohol.
Research conducted by Dr. McBride and colleagues reveals, for the first time, a common molecular mechanism for aortic valve stenosis, coarctation of the aorta and hypoplastic left heart syndrome.
Research from Nationwide Children’s Hospital is providing clues to the genetic elements of autism spectrum disorders with findings that could play an important role in genetic counseling.
The PKU Expanded Access Program provides early access to drugs for kids with a rare genetic metabolic disorder.