Gene therapy targeting nerve cells in the gut improved nerve cell survival and intestinal function — specifically that of the bowel — while also increasing survival rates in a mouse model of Batten disease, a new study by researchers at Washington University School of Medicine (WashU Medicine) in St.

Researchers have developed a mouse model of juvenile Batten disease that better reflects the survival outcomes of human patients, according to a new study. While mice without the gene that carries instructions for the CLN3 protein recapitulate many juvenile Batten features, these mice have milder disease and a much…

A treatment using stem cells modified to produce PPT1 — the missing enzyme in infantile Batten disease — prevented symptoms from developing and substantially extended survival in a mouse model of the disease, a study reports. It also prolonged survival and slowed disease progression when given to mice that…

Researchers described the natural history of retinal degeneration in a mouse model of CLN7, a form of late-infantile Batten disease, that mimics human patient retinal disease progression. The study revealed a progressive loss or dysfunction of various types of nerve cells within the retina that relay signals from the…

CLN5 disease, a form of late infantile Batten disease, disrupts fatty molecule balance and autophagy (the waste disposal system of cells), and increases cellular susceptibility to a type of damage called oxidative stress, according to data from preclinical models of the disease. Also, many of the molecules whose levels…

Drinking acidified water prevented motor impairment and decreased the toxic accumulation of molecular debris in brain cells in a mouse model of CLN1 disease, also called infantile Batten disease, a recent study reports. “In this study, we demonstrated that acidified drinking water had beneficial effects in … a mouse…

Six months of treatment with cannabidiol (CBD) reduced brain inflammation in a mouse model of infantile Batten disease, but did not affect the frequency of seizures or the survival of nerve cells, a new study reports. Given the severe symptoms that typically characterize this subtype of Batten disease,…

A screening of U.S.-approved therapies revealed that tamoxifen, an oral cancer therapy, is able to effectively reduce the accumulation of a fatty molecule called globotriaosylceramide, or Gb3, in cellular models of two forms of Batten disease. It worked without compromising cell survival, a study showed. That study found that…

A gene therapy designed to deliver a working version of the CLN8 gene — the one mutated in people with CLN8, a form of late-infantile Batten disease — prolonged the survival, reduced disease-associated brain features, and partially corrected motor problems in a mouse model. The findings, likely representing one of…

A single injection into the brain of an investigational gene therapy now in clinical trials partly prevented retinal degeneration and vision loss in a mouse model of CLN6 Batten disease, a study shows. These findings mark the first report of a brain-delivered gene therapy reaching both the brain and…