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Gene therapy may be classified into two types by the type of cell it affects: somatic cell and germline gene therapy. In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete , germ cell , gametocyte , or undifferentiated stem cell .
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
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The therapy known as Casgevy [11] works through editing a dysfunctional protein that interferes with creation of adult hemoglobin. This gene is known as the BCL11A, and when people have Beta thalassemia, their bodies do not make enough adult hemoglobin. Casgevy uses precise gene editing of stem cells, and reduces the activity of BCL11A.
How vectors work to transfer genetic material. Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by several methods, summarized below. The two major classes of methods are those that use recombinant viruses (sometimes called biological nanoparticles or viral vectors) and those that use naked DNA or DNA complexes (non-viral methods).
This is an accepted version of this page This is the latest accepted revision, reviewed on 17 November 2024. Manipulation of an organism's genome For a non-technical introduction to the topic of genetics, see Introduction to genetics. For the song by Orchestral Manoeuvres in the Dark, see Genetic Engineering (song). For the Montreal hardcore band, see Genetic Control. Part of a series on ...
The use of gene therapy in treating neurological disorders such as epilepsy has presented itself as an increasingly viable area of ongoing research with the primary targets being somatostatin, galanin, neuropeptide y, potassium channels, optogenetics and chemogenetics for epilepsy. As the field of gene therapy continues to grow and show ...
Logistics departments of biopharma companies experience new obstacles because of the introduction of new cell and gene therapy products, such as CAR T-cell therapies and allogeneic therapies. Cell and gene therapies require manufacturer and distributors alike to implement new systems and processes in order to ensure safe handling and delivery.