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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- Fall '17