University of Arizona Health Sciences immunobiologists have created a five-module chimeric antigen receptor T cell that is showing early potential to fight Type 1 diabetes.
Researchers at Indiana University School of Medicine have published their work about a specific type of childhood cancer in the peer-reviewed, international oncology journal, Cancers. This research involves a combination therapy that significantly slows tumor growth in models, which includes a model established from cells taken from tumors donated by Tyler Trent.
Researchers have identified two cell signals - Ccr5 and Cxcr6 - that are sent out by dying retinal cells to recruit stem cells and repair eye damage. When genetically engineered stem cells with an overabundance of Ccr5 and Cxcr6 cell receptors were transplanted into human and mouse models, they displayed a significantly higher rate of migration to degenerating retinal tissue, rescuing them from death and preserving their function.
Watch a video about Evie's treatment with an experimental gene therapy for sickle cell disease here: https://www.youtube.com/watch?v=XmQJpuLx07Y
Combining chimeric antigen receptor (CAR) T cell therapy with a PAK4 inhibitor drug allowed the engineered cells to punch their way through and attack solid tumors, leading to significantly enhanced survival in mice.
Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating an eye disease that leads to a progressive loss of vision and affects thousands of people across the globe. The study, which involved a collaboration with clinical teams in the Royal Victoria Eye and Ear Hospital and the Mater Hospital, also has implications for a much wider suite of neurological disorders associated with ageing.
Researchers at Emory and Case Western Reserve have re-engineered an oncolytic adenovirus. The resulting virus is not easily caught by parts of the innate immune system, making systemic delivery possible without arousing a massive inflammatory reaction.
In a promising advance for affordable, personalised medicine, researchers have used metal-organic frameworks to successfully deliver the genetic snipping tool CRISPR/Cas9 into human cancer cells.
In a comprehensive analysis of patients with cancer who had exceptional responses to therapy, researchers have identified molecular changes in the patients' tumors that may explain some of the exceptional responses. The results demonstrate that genomic characterizations of cancer can uncover genetic alterations that may contribute to unexpected and long-lasting responses to treatment, according to the researchers. The results appeared in Cancer Cell on Nov. 19, 2020.
Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH).