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Gene Therapy

4,076 bytes added, 22:13, 29 November 2018
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<p><a href="https://en.wikipedia.org/wiki/Genome_editing" title="Genome editing">Gene editing</a>&nbsp;is a potential approach to alter the human genome to treat genetic diseases,<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-34">[34]</a></sup>&nbsp;viral diseases,<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-35">[35]</a></sup>&nbsp;and cancer.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-36">[36]</a></sup>&nbsp;As of 2016&nbsp;these approaches were still years from being medicine.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-37">[37]</a></sup><sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-38">[38]</a></sup></p>
 
<h2>Vectors</h2>
 
<p>Main article:&nbsp;<a href="https://en.wikipedia.org/wiki/Vectors_in_gene_therapy" title="Vectors in gene therapy">Vectors in gene therapy</a></p>
 
<p>The delivery of DNA into cells can be accomplished by multiple&nbsp;<a href="https://en.wikipedia.org/wiki/Vectors_in_Gene_Therapy" title="Vectors in Gene Therapy">methods</a>. The two major classes are&nbsp;<a href="https://en.wikipedia.org/wiki/Recombinant_virus" title="Recombinant virus">recombinant viruses</a>&nbsp;(sometimes called biological nanoparticles or viral vectors) and&nbsp;<a href="https://en.wikipedia.org/wiki/Naked_DNA" title="Naked DNA">naked DNA</a>&nbsp;or DNA complexes (non-viral methods).</p>
 
<h3>Viruses</h3>
 
<p>Main article:&nbsp;<a href="https://en.wikipedia.org/wiki/Viral_vector" title="Viral vector">Viral vector</a></p>
 
<p>In order to&nbsp;<a href="https://en.wikipedia.org/wiki/Viral_replication" title="Viral replication">replicate</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Virus" title="Virus">viruses</a>&nbsp;introduce their genetic material into the host cell, tricking the host&#39;s cellular machinery into using it as blueprints for viral proteins.&nbsp;<a href="https://en.wikipedia.org/wiki/Retrovirus" title="Retrovirus">Retroviruses</a>&nbsp;go a stage further by having their genetic material copied into the genome of the host cell. Scientists exploit this by substituting a virus&#39;s genetic material with therapeutic DNA. (The term &#39;DNA&#39; may be an oversimplification, as some viruses contain RNA, and gene therapy could take this form as well.) A number of viruses have been used for human gene therapy, including&nbsp;<a href="https://en.wikipedia.org/wiki/Retrovirus" title="Retrovirus">retroviruses</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Adenovirus" title="Adenovirus">adenoviruses</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Herpes_simplex_virus" title="Herpes simplex virus">herpes simplex</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Vaccinia_virus" title="Vaccinia virus">vaccinia</a>, and&nbsp;<a href="https://en.wikipedia.org/wiki/Adeno-associated_virus" title="Adeno-associated virus">adeno-associated virus</a>.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-JGenMed_Database-4">[4]</a></sup>&nbsp;Like the genetic material (DNA or RNA) in viruses, therapeutic DNA can be designed to simply serve as a temporary blueprint that is degraded naturally or (at least theoretically) to enter the host&#39;s genome, becoming a permanent part of the host&#39;s DNA in infected cells.</p>
 
<h3>Non-viral</h3>
 
<p>Non-viral methods present certain advantages over viral methods, such as large scale production and low host&nbsp;<a href="https://en.wikipedia.org/wiki/Immunogenicity" title="Immunogenicity">immunogenicity</a>. However, non-viral methods initially produced lower levels of&nbsp;<a href="https://en.wikipedia.org/wiki/Transfection" title="Transfection">transfection</a>&nbsp;and&nbsp;<a href="https://en.wikipedia.org/wiki/Gene_expression" title="Gene expression">gene expression</a>, and thus lower therapeutic efficacy. Later technology remedied this deficiency.<sup>[<em><a href="https://en.wikipedia.org/wiki/Wikipedia:Citation_needed" title="Wikipedia:Citation needed">citation needed</a></em>]</sup></p>
 
<p><a href="https://en.wikipedia.org/wiki/Vectors_in_Gene_Therapy#Non-viral_methods" title="Vectors in Gene Therapy">Methods for non-viral gene therapy</a>&nbsp;include the injection of naked DNA,&nbsp;<a href="https://en.wikipedia.org/wiki/Electroporation" title="Electroporation">electroporation</a>, the&nbsp;<a href="https://en.wikipedia.org/wiki/Gene_gun" title="Gene gun">gene gun</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Sonoporation" title="Sonoporation">sonoporation</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Magnetofection" title="Magnetofection">magnetofection</a>, the use of&nbsp;<a href="https://en.wikipedia.org/wiki/Oligonucleotide" title="Oligonucleotide">oligonucleotides</a>, lipoplexes, dendrimers, and inorganic nanoparticles.</p>
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