Emory researchers used CRISPR/Cas9 gene editing to snip part of a gene producing toxic Huntington's disease protein aggregates in the brains of 9-month-old mice. Weeks later, where the delivery vehicle was applied, aggregated proteins had almost disappeared. In addition, the motor abilities of the mice had improved, although not to the level of control mice.
Gene transfer with laboratory-produced viruses is seen as a hopeful therapy for Alzheimer's and Parkinson's patients. A team from Vetmeduni Vienna investigated how far these viruses spread in the brain and which cells they infect. Some of these viruses travelled from injection site as far as the olfactory bulb or the cerebellum and infected neurons and other cells. This could improve selection of viral vectors for custom therapies using gene transfer. Histochemistry and Cell Biology
Scientists at the Biogerontology Research Foundation, Feinberg School of Medicine at Northwestern University and Swammerdam Institute of Life Sciences at the University of Amsterdam have published a paper on a proposed method of in situ tissue regeneration called Induced Cell Turnover in the journal Human Gene Therapy.
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response.
A new study of gene transfer using adeno-associated virus (AAV)-based gene delivery into skeletal muscle of rhesus macaques showed that oral prednisone reduced immune responses to AAV that can weaken expression of the therapeutic transgene over time.
Based on the encouraging safety data that has emerged from multiple clinical trials that used different gene transfer approaches to improve heart function in patients suffering from heart failure with reduced ejection fraction, researchers conclude that this therapeutic strategy can be advanced with acceptable risk.
An IDIBELL research team manages to characterize the complete epigenomes of the most frequent tumors, including those of colon, lung and breast cancer. The study represents a big step in the study of origin and progression of these tumors.
Whether using embryonic or adult stem cells, coercing these master cells to convert to the desired target cell and reproduce flawlessly is difficult. Now an international team of researchers has a two-part system that can convert the cells to the targets and then remove the remnants of that conversion, leaving only the desired DNA behind to duplicate.
A team of researchers at the University of Central Florida is using breakthrough gene-editing technology to develop a new screening tool for Parkinson's disease, a debilitating degenerative disorder of the nervous system. The technology allows scientists in the lab to 'light up' and then monitor a brain protein called alpha-synuclein that has been associated with Parkinson's.
Triggered by decreases in ER Ca2+ level, conformational changes of the endoplasmic reticulum (ER) luminal domain in STIM1 would switch on its cytoplasmic domain (CT) to engage and gate ORAI channels on plasma membrane, resulting in Ca2+ influx.