A Dual Gene Therapy and Gene Editing Platform
May 14, 2020 | Biotechnology
Reading time: 4-5 minutes
Today, Lab.Equipment has curated a new Biotechnology article published from Genetic Engineering and Biotechnology News (GEN) just for you:
Advances in gene therapy and gene editing represent the future for many rare diseases where a single administration may lead to durable and potentially curative treatments. These two approaches share a similar basis, but they are distinct technologies. In specific contexts, each may present different advantages.
Gene therapy aims to deliver functional copies of the causal gene to the exact location in the body where the disease manifests. Gene therapies reside inside the nucleus of target organ cells as standalone DNA constructs and do not integrate into the genome. These constructs can be lost as cells divide. Yet the constructs have the potential to cure those diseases where the target organ is comprised of slowly dividing or nondividing cells, such as the cells of the adult liver or central nervous system.
Conversely, the goal of gene editing is to insert a functional gene directly into the genome of an affected patient. In doing so, gene editing is designed to provide a permanent correction that may be maintained even as cells divide. This is achieved by designing the gene editing construct to specifically target a certain region of the genome. Then, by using the body’s natural DNA repair process, called homologous recombination, a functional gene can be inserted. Figure 1 shows the key differentiators between gene therapy and gene editing approaches... More of this in the GEN – Genetic Engineering and Biotechnology News Blog.
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