News by Subject Biology

Isabelle Richard's team, a CNRS researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has demonstrated the efficacy of gene therapy and determined the effective dose for treating a rare muscle disease, gamma-sarcoglycanopathy, in mouse models of the disease. Based on these encouraging results, published in Molecular Therapy: Methods and Clinical Development, the researchers are preparing a clinical trial.

Adding genomic sequencing results to traditional newborn screening means a baby could potentially test positive for numerous conditions that might not develop within their lifetime. A UNC School of Medicine study proposed a method for how to responsibly determine which types of conditions to include in testing and potentially return to parents.

The study shows that tumour suppressor hnRNP K can lead to cancer. Overexpression of the gene may lead to B-cell lymphoma, the most common type of blood cancer. Lymphoma patients might benefit from more personalised treatments.

Professor Yong-Seok Oh's team at the DGIST Department of Brain-Cognitive Science clarified the expression of antidepressant efficacy by modulating hippocampal mossy cells. Expects to provide a basis to understand the mechanism of existing anti-depressants and contribute greatly to the development of next generation depression treatment.

Autism spectrum disorder (ASD) is highly variable disorder, both in its presentation and in its genetics -- hundreds of risk genes have been identified. One key to understanding and ultimately treating ASD is to identify common molecular mechanisms underlying this genetically heterogeneous disorder. Four Canadian researchers presented the results of unique approaches to understand ASD at the 14th Canadian Neuroscience Meeting in Toronto, on May 24, 2019.

New research on amyotrophic lateral sclerosis (AML) has revealed that a protein called membralin plays a key role in the disease process. The study, published in Journal of Clinical Investigation, suggests that membralin-boosting gene therapy is a potential therapeutic direction to treat this often deadly disease.

CRISPR/Cas9 technology enables convenient and effective genome editing in diploid cell lines based on the isolation and expansion of edited single-cell clones. However, this approach is ineffective for aneuploid cell lines, and a group has now reported an improved method for genome editing based on multiple rounds of modification.

A close look at the rapidly developing zebrafish embryo is helping neuroscientists better understand the potential underpinnings of brain disorders, including autism and schizophrenia. The new study, published online this month in the journal eNeuro, points to a 'clustering' of cellular interactions in the brain that may disrupt normal development and brain health.