Gene therapy
Researchers from Najran University have published a review highlighting the potential of CRISPR-Cas9 gene therapy in treating sickle cell disease (SCD) and β-thalassemia (BT). According to the study, SCD and BT affect millions of people worldwide, with approximately 500,000 infants born with SCD and 60,000 people diagnosed with
Genetic research
Scientists at The University of Osaka have made a groundbreaking discovery in the field of gene therapy, uncovering the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors. The findings, published in a recent study, reveal that the N-terminal region of the VP1 protein undergoes structural changes upon heating,
Gene therapy
Scientists at the Hubrecht Institute, Erasmus MC, and Sanquin have made a groundbreaking discovery in gene therapy by finding a way to restart inactive genes using a new approach called "delete-to-recruit." This innovative method uses CRISPR-Cas9 technology to cut out a piece of DNA between an enhancer and
Cancer
Researchers from King Saud University in Riyadh, Saudi Arabia, have made significant strides in cancer gene therapy, highlighting the potential of subcellular delivery with a focus on mitochondrial pharmacology, gene therapy, transplantation, and drug targeting. The study leverages scholarly sources, including PubMed, Google Scholar, and Scopus, to emphasize the importance
Genetic research
Gene therapy has shown remarkable results in restoring hearing in toddlers and young adults born with congenital deafness, specifically in those with OTOF-related deafness. A study conducted by researcher Maoli Duan and their team has demonstrated that the therapy can be safely administered to patients of various ages, including those
Genetic research
Investigators at Tungs' Taichung MetroHarbor Hospital in Taichung, Taiwan, have made groundbreaking discoveries in cancer gene therapy using the oncolytic avian reovirus (ARV) p17 protein. According to a recent study published in Frontiers in Cellular and Infection Microbiology, ARV p17 protein shows significant potential in inhibiting cancer cell migration
Cancer
Scientists at Lomonosov Moscow State University have made significant advancements in the development of sorafenib delivery systems, a proven treatment for kidney cancer, to improve its bioavailability and expand its therapeutic potential to other oncological diseases. By leveraging biopolymers and supramolecular assemblies, researchers have created more sophisticated delivery systems with
Cancer
Researchers from Chuo University in Tokyo, Japan, have made a significant breakthrough in cancer gene therapy by developing micro/nano motors that can deliver targeted drugs to cancer cells. These self-propelled micro motors, functionalized with doxorubicin-loaded liposomes, can be triggered to release the drug upon near-infrared irradiation, leading to efficient
Genetic research
Scientists at Hokkaido University have made a groundbreaking discovery in the field of gene therapy, developing a novel method for delivering the CRISPR/Cas9 system directly into the mitochondria of cells. The innovative approach, known as RNP-MITO-Porter, utilizes a lipid nanoparticle delivery system to target a point mutation in mitochondrial
Genetic research
Research from Shanghai Jiao Tong University School of Medicine has shed new light on gene therapy for thalassemia, a genetic disorder that affects the production of hemoglobin in red blood cells. According to a recent study published in the Shanghai Jiaotong Daxue xuebao. Yixue ban journal, traditional treatment modalities for
Cancer
Research conducted at the University of Science and Technology of China has revealed significant advancements in cancer gene therapy using adeno-associated virus (AAV) vectors. AAV vectors have been recognized as a safe and effective method for delivering therapeutic transgenes, with four AAV-based gene therapy drugs already approved by the US
Genetic research
Gene therapy researchers at Miltenyi Biotec GmbH have made significant advancements in optimizing CAR-gd T cell therapies for enhanced anti-tumor potency. The team, led by Lorraine Pinot and including Aylin Saßor, Nina Moker, Congcong Zhang, Els Verhoeyen, Jose Villacorta Hidalgo, and Rimas J. Orentas, employed a Baboon-pseudotyped lentiviral vector (BaEV-LV)
