Lecture 21 Flashcards
What is gene therapy?
It is adding a corrected copy of a defective gene
How is gene therapy done?
It normally works through a virus adding/delivering a gene to cells where it is needed
What are good candidates for gene therapy?
Genetic disorders caused by mutations in the single gene. Those that are caused by multiple genes not so much
What is true about gene therapy?
You need to know which gene is affected.
How can you restore the CFTR protein?
Gene transfer because it adds a normal CFTR DNA seqeunce. This can be given through inhaling and IV
Is CF a good candidate for gene therapy?
Yes because it involved one gene which is chromosome 7
What is cystic fibrosis?
A condition that regulated the flow of salt in cells. However, in the condition, this is disrupted and causes build up of mucus and affects lungs, pancreas, and small intestines.
What are some challenges in CF and gene therapies
Inhalation and IV are used. Inhalation is hard because there is the build up of mucus while IV has to pass through multiple layers of tissues
What is a vector?
They are like vehicles that deliver the genetic material needed for the cells
How can gene therapy restore the function of CFTR?
When delivered through inhaling, they help restore function in the lungs. The cells can create their own CF protein to not create the mucus build up.
What therapy can be used to make CFTR protein?
DNA or RNA therapy
What is cell based therapy?
It is removing cells in the airway to create space
What is transformation?
It is the process where a cell(usually bacteria) can take up DNA from their environment and use it
What is genetic engeneering?
Changing an organism’s genotype using biotech tools or technique
What are restriction enzymes?
Enzymes that cut in specific spots. These are used in plasmids during genetic engineering.
What is a plasmid? What is it used for?
Extra genes that can be used
How has gene therapy helped in disorders?
It has helped restore the normal functions. This was tested in dog patients.
Who was Jesse Gelsinger?
He was a volunteer in the therapy trial and had an illness in the liver. He received the therapy but instead his body rejected it and attacked his organs
