Gene Therapy

Rice biomolecular engineers' tool sets precisely fix cytosine mutations in human genomeCredit: Gao Lab/Rice University HOUSTON - (July 15, 2020) - Biomolecular engineers at Rice University have found a C-worthy technique…

Peer reviewed - experimental study - people and animalsCredit: Alex Taylor Researchers at the University of East Anglia and University of Manchester have made an important breakthrough that could lead to 'kinder'…

Editing technique repairs mutant MeCP2 RNA and restores MeCP2 protein functionCredit: Rett Syndrome Research Trust A genomic error that causes Rett Syndrome, a serious lifelong neurological disorder, can be corrected in the brains…

Credit: UVA Health Scientists have identified an oncogene (a cancer-causing gene) responsible for glioblastoma, the deadliest brain tumor. The discovery offers a promising new treatment target for a cancer that is always fatal. The…

DNA hypermethylation responsible for reduced amygdala volume in male patients with schizophrenia and bipolar disorder.A) CpG site where the structure and methylation of the SERT gene were analyzed. Transcription is performed from right to…

Findings could lead to new therapies for cancer, heart abnormalities Look deep inside our cells, and you'll find that each has an identical genome -a complete set of genes that provides the instructions for our cells' form and function.…

Credit: Mary Ann Liebert, Inc., publishers New Rochelle, NY, July 2, 2020--The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy…

Controlling the clearance of gene therapy drugs at the liver sinusoidal wall 5pm on June 26, 2020 - Kawasaki/Japan: The Innovation Center of NanoMedicine (iCONM), the National Institute for Quantum Science and Technology (QST), and…

A new test developed at CHU Sainte-Justine in Montreal will enable better management of patients with severe combined immunodeficiency (SCID). The results of this work was presented Tuesday in the medical journal Blood Advances published…

University of Iowa team makes gene therapy approach freely available for COVID-19 researchCredit: University of Iowa Health Care Until there are effective treatments or vaccines, the COVID-19 pandemic will remain a significant…

NIH effort expands data collection to shed light on pandemic's spread and impactCredit: National Institutes of Health The All of Us Research Program, part of the National Institutes of Health, today announced that it is leveraging…

UCLA researchers and colleagues have received a $13.65 million grant from the National Institutes of Health to investigate and further develop an immunotherapy known as CAR T, which uses genetically modified stem cells to target and…

A mouse model and previous studies suggest that genetic intervention in SHANK3-related ASD may be most effective earlier in developmentCredit: UAB BIRMINGHAM, Ala. - A mouse study by Craig Powell, M.D., Ph.D., and colleagues suggests…

St. Jude Children's Research Hospital software enables detection of previously unknown cancer-causing gene fusions, pointing the way to new treatments. After years of development, engineering and enhancement, researchers at St. Jude…

Credit: IU School of Medicine More than 3 million Americans have glaucoma, a serious eye condition causing vision loss. Using human stem cell models, researchers at Indiana University School of Medicine found they could analyze…

Scientists have developed a new gene therapy approach that offers tremendous promise for one day treating an eye disease that leads to blindness and affects thousands of people across the globe. Researchers from Trinity College Dublin…

UCLA team's approach suggests possible path toward treating other severe infectionsCredit: Nick Carranza / UCLA Health In the June 11 issue of the New England Journal of Medicine, a team of UCLA physicians and scientists describes…

These important proof-of-concept studies could one day lead to new real-time diagnostic imaging techniques and the identification of genomic biomarkers that predict treatment results -- innovations that will improve health outcomes for…

Credit: RCSI Scientists have found that a specific type of ovarian cancer could possibly benefit from existing platinum-based chemotherapy and new DNA repairing treatments, following better testing. The study, led by researchers…

This result opens up new therapeutic prospects and the possibility of treating more patients. A research team from Genethon, in collaboration with teams from CNRS/Inserm and from the biotechnology company Spark Therapeutics, announced…