Tag: Gene Therapy

The fight against cystic fibrosis (CF) has taken a major step forward, with pioneering research by University of Adelaide scientists showing that cells causing the debilitating genetic disorder could be successfully replaced with healthy ones. The research published in the journal Stem Cell Research and Therapy applies cell transplantation therapy, normally used in bone marrow

NIH-funded researchers delayed signs of amyotrophic lateral sclerosis (ALS) in rodents by injecting them with a second-generation drug designed to silence the gene, superoxide dismutase 1 (SOD1). The results, published in the Journal of Clinical Investigation, suggest the newer version of the drug may be effective at treating an inherited form of the disease caused

A City of Hope scientist has discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases, positioning the new tool as the basis for the next generation of genetic therapies. This editing platform, discovered by City of Hope’s Saswati Chatterjee, Ph.D., eventually may be used to cure inherited

Researchers from the University of Illinois at Chicago are the first to describe why CRISPR gene editing sometimes fails to work, and how the process can be made to be much more efficient. CRISPR is a gene-editing tool that allows scientists to cut out unwanted genes or genetic material from DNA, and sometimes add a

Researchers have developed a 3D map of the gene interactions that play a key role in cardiovascular disease, a study in eLife reports. The map will help researchers identify the most important genes to focus on for the development of new treatments for heart attacks, heart failure and heart rhythm disorders. More than 500 genetic

How are chromosomes arranged in the cell nucleus? Is it possible that they communicate with one another by “touching” each other? To answer this question and to shed more light on the fundamental properties of the communication between different chromosomes, Philipp Maass from Friedrich Luft’s lab at the Max Delbrück Center for Molecular Medicine in

New preclinical research shows a gene already linked to a subset of people with autism spectrum disorder is critical to healthy neuronal connections in the developing brain, and its loss can harm those connections to help fuel the complex developmental condition. Scientists at Cincinnati Children’s Hospital Medical Center report in Developmental Cell their data clarify

Pregnancy loss and the death of a newborn baby are devastating events, and as of today around 25% of these perinatal deaths are unexplained despite autopsy. Discovering the cause of such a loss is of great importance for the bereaved parents, both in providing an explanation and in helping them to understand the likelihood of

Researchers at the RIKEN Center for Brain Science in Japan have discovered that a gene associated with human intellectual ability is necessary for normal memory formation in mice. Published in Nature Neuroscience, the study shows that mice with only one copy of the gene replay shorter fragments of their previous experiences during periods of rest,

In-home rehabilitation, using a telehealth system and supervised by licensed occupational/physical therapists, is an effective means of improving arm motor status in stroke survivors, according to findings presented by University of California, Irvine neurologist Steven C. Cramer, MD, at the recent 2018 European Stroke Organisation Conference in Gothenburg, Sweden. “Motor deficits are a major contributor

Researchers from Columbia University have developed a new technique for the powerful gene editing tool CRISPR to restore retinal function in mice afflicted by a degenerative retinal disease, retinitis pigmentosa. This is the first time researchers have successfully applied CRISPR technology to a type of inherited disease known as a dominant disorder. This same tool

Attempted vaginal birth after cesarean (VBAC) is associated with higher rates of adverse effects or death for mothers and infants, although absolute rates were low in mothers who attempted this type of birth, according to research in CMAJ (Canadian Medical Association Journal). “The absolute rates of adverse maternal and neonatal outcomes are low with both

Biomedical engineers at Duke University have used a CRISPR/Cas9 genetic engineering technique to turn off a gene that regulates cholesterol levels in adult mice, leading to reduced blood cholesterol levels and gene repression lasting for six months after a single treatment. This marks the first time researchers have delivered CRISPR/Cas9 repressors for targeted therapeutic gene

Although cannabis, also known as marijuana, has been shown to impair memory, reaction time and attention, it is difficult to assess this impairment in a natural setting. Researchers have developed a prototype app called ‘Am I Stoned’ that could help cannabis users understand how the drug is affecting them through a series of phone-based tasks.

Scientists from the Institut Pasteur set out to understand how the demographic changes associated with the Neolithic transition also influenced the efficacy of natural selection. By comparing the genome diversity of more than 300 individuals from groups of forest hunter-gatherers (pygmies) and farmers (Bantu-speaking peoples), from western and eastern Central Africa, they discovered that the