Every virus system has advantages and disadvantages as a vector, depending on its intended use. One of the more exciting uses has been the development of viruses as vectors for gene therapy, that is, to correct genetic defects in humans. In the most general sense, gene therapy involves ...
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 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 chemotherapy or viral infection. While gene th...
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...
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.
Various long-term treatments for anaemia, haemophilia, CF , dystrophy , Gauscher’s disease, lysosomal storage diseases, cardiovascular diseases, diabetes and diseases of bones and joints are resolved through successful gene therapy Related Conferences: Genetics Conferences | Genetics 2023 | Genetics ...
Fig. 1: Collagen VII (C7) expression in primary RDEB patient cells, RDEB mice, and human RDEB xenografts on immunodeficient mice following B-VEC therapy. a, RDEB keratinocytes and fibroblasts were infected in vitro with B-VEC vector at various ratios of cell to viral particle (MOI). Cells...
Parrish says she had received two forms of gene therapy produced under contract with a commercial laboratory, which she did not identify, outside the United States. In one treatment, she says, she received injections into her muscles containing the gene follistatin, which in animal experiments is...
Anti-CD19 chimeric antigen receptor (CAR) T cell therapy is a common, yet highly efficient, cellular immunotherapy for lymphoma. However, many recent studies have reported on its cardiovascular (CV) toxicity. ... Yang Liu, Xiaoshuang Yuan, Xu Yang, Bo Yang, Guangyang Liu, Xiao Xu, Sanbin...
We then summarize the progress made in applying genome editing to various areas of gene and cell therapy, including antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders. The current challenges and future prospects for genome editing as a transformative technology for...