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Gene Therapy

4,251 bytes added, 22:11, 29 November 2018
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<p>Not all medical procedures that introduce alterations to a patient&#39;s genetic makeup can be considered gene therapy.&nbsp;<a href="https://en.wikipedia.org/wiki/Bone_marrow_transplantation" title="Bone marrow transplantation">Bone marrow transplantation</a>&nbsp;and&nbsp;<a href="https://en.wikipedia.org/wiki/Organ_transplants" title="Organ transplants">organ transplants</a>&nbsp;in general have been found to introduce foreign DNA into patients.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-5">[5]</a></sup>&nbsp;Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effect.</p>
 
<h2>Approaches[<a href="https://en.wikipedia.org/w/index.php?title=Gene_therapy&amp;action=edit&amp;section=2" title="Edit section: Approaches">edit</a>]</h2>
 
<p>Following early advances in&nbsp;<a href="https://en.wikipedia.org/wiki/Genetic_engineering" title="Genetic engineering">genetic engineering</a>&nbsp;of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered &ndash; replacing or disrupting defective genes.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-28">[28]</a></sup>&nbsp;Scientists focused on diseases caused by single-gene defects, such as&nbsp;<a href="https://en.wikipedia.org/wiki/Cystic_fibrosis" title="Cystic fibrosis">cystic fibrosis</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Haemophilia" title="">haemophilia</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Muscular_dystrophy" title="Muscular dystrophy">muscular dystrophy</a>,&nbsp;<a href="https://en.wikipedia.org/wiki/Thalassemia" title="Thalassemia">thalassemia</a>, and&nbsp;<a href="https://en.wikipedia.org/wiki/Sickle_cell_anemia" title="Sickle cell anemia">sickle cell anemia</a>.&nbsp;<a href="https://en.wikipedia.org/wiki/Glybera" title="Glybera">Glybera</a>&nbsp;treats one such disease, caused by a defect in&nbsp;<a href="https://en.wikipedia.org/wiki/Lipoprotein_lipase" title="Lipoprotein lipase">lipoprotein lipase</a>.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-Gallagher-27">[27]</a></sup></p>
 
<p><a href="https://en.wikipedia.org/wiki/DNA" title="DNA">DNA</a>&nbsp;must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-29">[29]</a></sup>&nbsp;Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered&nbsp;<a href="https://en.wikipedia.org/wiki/Virus" title="Virus">virus</a>&nbsp;to deliver the DNA into a&nbsp;<a href="https://en.wikipedia.org/wiki/Chromosome" title="Chromosome">chromosome</a>.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-pmid23015375-30">[30]</a></sup><sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-pmid23435812-31">[31]</a></sup>&nbsp;<a href="https://en.wikipedia.org/wiki/Naked_DNA" title="Naked DNA">Naked DNA</a>&nbsp;approaches have also been explored, especially in the context of&nbsp;<a href="https://en.wikipedia.org/wiki/Vaccine" title="Vaccine">vaccine</a>&nbsp;development.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-pmid23111168-32">[32]</a></sup></p>
 
<p>Generally, efforts focused on administering a gene that causes a needed protein to be expressed. More recently, increased understanding of&nbsp;<a href="https://en.wikipedia.org/wiki/Nucleases" title="Nucleases">nuclease</a>&nbsp;function has led to more direct DNA editing, using techniques such as&nbsp;<a href="https://en.wikipedia.org/wiki/Zinc_finger_nucleases" title="Zinc finger nucleases">zinc finger nucleases</a>&nbsp;and&nbsp;<a href="https://en.wikipedia.org/wiki/CRISPR" title="CRISPR">CRISPR</a>. The vector incorporates genes into chromosomes. The expressed nucleases then knock out and replace genes in the chromosome. As of 2014&nbsp;these approaches involve removing cells from patients, editing a chromosome and returning the transformed cells to patients.<sup><a href="https://en.wikipedia.org/wiki/Gene_therapy#cite_note-pmid20717154-33">[33]</a></sup></p>
 
<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>
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