Gene Therapy Notes Biomedical.pdf - Gene Therapy Notes \u25cf Experimental\u200b technique \u25cf Uses genes to prevent disease \u25cf May allow inserting a gene

Gene Therapy Notes Biomedical.pdf - Gene Therapy Notes...

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Gene Therapy Notes Experimental technique Uses genes to prevent disease May allow inserting a gene into cells instead of drugs or surgery Replace a mutated gene with a healthy copy Inactivating mutated gene Introducing new gene to fight a disease Gene Therapy targets somatic cells and changes will not be passed onto future generations. Challenges Condition must be well understood Underlying causative gene identified Means of efficiency delivering working copies to genes to cells -- Most difficult Vectors - Something that can carry a gene into a cell Some can insert the gene into cells DNA Can remove the virus gene 1 Virus can deliver gene to cell Transcripted gene can issue instructions to make correct protein A gene that is inserted directly into a cell usually does not function Virus modified so they can cause disease Retroviruses , integrate there genetic material (including the new gene) into a chromosome in the human cell Adenoviruses , introduce their DNA into the nucleus of the cell, but the DNA is not integrated into chromosome Vectors can be injected, or given intravenously (IV) directly into a specific tissue in the body where it is taken up by individual cells A sample of the patient's cells can be removed and exposed to the vector in a laboratory setting. The
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