"Recently, we designed a new gene therapy vector, and by introducing ournew therapyto the brains of mice via this vector, we have been able to show we can increase production of p38y only where it is needed." When treated with the new gene therapy, mice with uncontrolled epilepsy had a...
It's hoped that this type of therapy could reduce infected individuals' dependence on antiviral medications, lower the cost of therapy, and permit the possible eradication of HIV from its hiding places in the body, the researchers added.
Gene therapy utilizes nucleic acid drugs to treat diseases, encompassing gene supplementation, gene replacement, gene silencing, and gene editing. It represents a distinct therapeutic approach from traditional medications and introduces novel strategies for genetic disorders. Over the past two decades, sign...
Gene therapy may constitute a promising alternative to conventional pharmacological tools and surgeries for epilepsy. For primary epilepsy, a single variant leading to a significant effect is relatively rare, while other forms are considered complex in inheritances with multiple susceptible mutations and ...
Gene therapy is a rapidly growing field, but is hindered by efficacy and safety concerns, including those related to delivery methods. Here, inspired by the use of foam in the delivery of pharmaceuticals, Dr. Stephan and colleagues formulated foam as a safe and effective delivery platform for ...
Gene therapy can be used to restore cell function in monogenic disorders or to endow cells with new capabilities, such as improved killing of cancer cells, expression of suicide genes for controlled elimination of cell populations, or protection against
(small interfering RNA), shRNA (short hairpin RNA) and miRNA (microRNA) silencing the mutated gene by altering its expression on mRNA level. The last strategy is known as antisense therapy but it is appliable only when causative mutation is displayed as dominant negative or gain-of-function ...
The CRISPR system is a revolutionary genome editing tool that has the potential to revolutionize the field of cancer research and therapy. The ability to precisely target and edit specific genetic mutations that drive the growth and spread of tumors has
Clinical development of chimeric antigen receptor (CAR)-T-cell therapy has been enabled by advances in synthetic biology, genetic engineering, clinical-grade manufacturing, and complex logistics to distribute the drug product to treatment sites. A key am
Republic of Korea; 2Advanced Therapy Product Research Division, Ministry of Food and Drug Safety, Cheongju, Chungcheongbuk-do, Republic of Korea Abstract Gene-editing technology is an emerging therapeutic modality for manipulating the eukaryotic genome by using target-sequence- specific engineered nucleas...