May 30, 2018
…example of an effective gene therapy for deadly bacterial infections. In a paper recently published in the journal Nature Communications, UC San Diego Department of Chemistry and Biochemistry’s Michael Sailor, along with colleagues from the Sanford Burnham Prebys Medical Discovery Institute (SBP), the University of Minnesota and the Korea Advanced…
May 11, 2021
…potential new type of gene therapy using novel CRISPR-based techniques. Working in fruit flies and human cells, research led by UC San Diego Postdoctoral Scholar Zhiqian Li in Division of Biological Sciences Professor Ethan Bier’s laboratory demonstrates that new DNA repair mechanisms could be designed to address the effects of…
March 22, 2016
…trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B. The Phase I/II open-label trial is sponsored by Baxalta Incorporated and will assess the safety and optimal dosing level of an investigational blood clotting factor IX gene therapy treatment.…
October 17, 2016
…targeting these cells with gene therapy designed to prevent their death might slow progression of the disease,” said Robert N. Weinreb, MD, director of both the Hamilton Glaucoma Center and Shiley Eye Institute, and a co-author of the 2015 Cell Death and Disease paper. Weinreb, with senior author Wonkyu Ju,…
April 21, 2017
…strategy was to use gene therapy to make the underlying mutations irrelevant, resulting in the preservation of tissue and vision.” The scientists tested their approach in two different mouse models of RP. In both cases, they found an abundance of reprogrammed cone cells and preserved cellular architecture in the retinas.…
May 9, 2022
…Medicine, report that a gene therapy that inhibits targeted nerve cell signaling effectively reduced neuropathic pain with no detectable side effects in mice with spinal cord or peripheral nerve injuries. The findings, published in the May 5, 2022 online issue of Molecular Therapy, represent a potential new treatment approach for…
March 10, 2021
A gene therapy for chronic pain could offer a safer, non-addictive alternative to opioids. Researchers at the University of California San Diego developed the new therapy, which works by temporarily repressing a gene involved in sensing pain. It increased pain tolerance in mice, lowered their sensitivity to pain and provided…
September 14, 2020
…one dose of RCas9 gene therapy can chew up toxic RNA and almost completely reverse symptoms in a mouse model of myotonic dystrophy. “Many other severe neuromuscular diseases, such as Huntington’s and ALS, are also caused by similar RNA buildup,” said senior author Gene Yeo, PhD, professor of cellular and…
May 2, 2022
…disrupts neural development. Using gene therapy tools to recover the gene’s function effectively rescued neural structure and function. Several neurological and neuropsychiatric diseases, including autism spectrum disorders (ASD) and schizophrenia have been linked to mutations in Transcription Factor 4 (TCF4), an essential gene in brain development. Transcription factors regulate when…
February 13, 2018
…Devaraj. “Compared to DNA-based gene therapy, in vivo expression of therapeutic proteins through the direct translation of mRNA offers much quicker response times and minimizes the risk of causing insertional mutagenesis (DNA mutations). Our mRNA translation regulation approach can greatly expand the toolbox of mRNA modification for therapeutic discoveries.” Devaraj…
