A new comprehensive natural history study about Amish nemaline myopathy (ANM) in the Old Order Amish population focuses on the promise of gene therapy for this lethal disorder. Amish nemaline myopathy (ANM) is an infantile-onset muscle disease linked to a mutation of the TNNT1 gene. The study summarizes genealogical records, clinical data, and molecular reports of one hundred and six ANM patients born between 1923 and 2017.

Experimental gene therapies have yielded promising results in early trials. But the drugs have left some patients wary, worried that success will not last.

A single administration of a therapeutic vector in mouse models cures type 2 diabetes and obesity in the absence of long-term side effects, researchers report. In healthy mice, the therapy prevents age-associated weight gain and insulin resistance and promotes healthy aging.

"By increasing BMP4, we can increase the metabolic rate, but we only see this in initially lean mice.

A new gene therapy that targets the heart and requires only one treatment session has been found safe for patients with coronary artery disease, according to a successful trial carried out in Finland.