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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.
Jesse Gelsinger (June 18, 1981 – September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver, the symptoms of which include an inability to metabolize ammonia – a byproduct of protein ...
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).
The first is alteration of germ cells, that is, sperm or eggs, which results in a permanent genetic change for the whole organism and subsequent generations. This "germ line gene therapy" is considered by many to be unethical in human beings. [22] The second type of gene therapy, "somatic cell gene therapy", is analogous to an organ transplant.
Gene knockdown is an experimental technique by which the expression of one or more of an organism's genes is reduced. The reduction can occur either through genetic modification or by treatment with a reagent such as a short DNA or RNA oligonucleotide that has a sequence complementary to either gene or an mRNA transcript.
The framework lacks the requisite international treaties for enforcement. At the first International Summit on Human Gene Editing in December 2015 researchers issued the first international guidelines. [16] These guidelines allowed pre-clinical research into gene editing in human cells as long as the embryos were not used to implant pregnancy.
Gene knockout by mutation is commonly carried out in bacteria. An early instance of the use of this technique in Escherichia coli was published in 1989 by Hamilton, et al. [2] In this experiment, two sequential recombinations were used to delete the gene. This work established the feasibility of removing or replacing a functional gene in bacteria.
The genetic insert was designed to only be expressed in S or M cones, and the expression of GFP in vivo was observed over time. Gene expression could stabilize if a sufficiently high dose of the viral vector is given. In 2009, adult dichromatic squirrel monkeys were converted into trichromats using gene therapy. [6]
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