What is point mutation?
A change in any single nucleotide of a DNA sequence.
What is deletion?
The loss of one or more nucleotides in a DNA sequence.
What is insertion?
The addition of one or more nucleotides in a DNA sequence.
What is a silent mutation?
If a point mutation doesn’t cause a change in the amino acid sequence.
What is a missense mutation?
A mutation that causes one amino acid in the sequence to change into a different one.
What is a nonsense mutation?
A mutation that results in a stop codon where there used to be an amino acid codon, resulting in the translation being stopped before the primary structure of the protein is complete.
What is artificial breeding?
Breeding for a specific desired trait.
Why is DNA considered universal?
The building blocks are found in all organisms, just different sequences.
Define biotechnology
The technological application of biology oriented to serve our life.
Define gene mutation
A alteration in the nucleotide sequence of an organism’s genome.
Define genome
The complete set of genes or genetic material present in a cell or organism.
What is the function of RBCs?
Transport oxygen from the lungs to all the tissues in the body
What proteins inside the RBCs carry oxygen?
Hemoglobin
Compare the hemoglobin shape of sickle and normal RBCs.
Hemoglobin in normal RBCs are donut-shaped, flexible, and float independently. Hemoglobin in sickle RBCs are aligned in rows, sickle-shaped, and are hard/sticky, causing them to block vessels.
What problems can sickle cells cause in the spleen?
Puts patients at risk for dangerous infections
What problems can sickle cells cause in the lungs?
Produce fevers and difficulty breathing
What problems can sickle cells cause in the eyes?
Vision problems and retinal dysfunction
What problems can sickle cells cause in the brain?
Strokes
How long do healthy and sickle RBCs live?
Healthy live 4 months, while sickled live 10-20 days.
What is anemia?
A condition of having less than the normal number of RBCs
What are stem cells?
Undifferentiated cells
What is gene therapy?
A potential solution to fix a genetic problem at its source by adding a corrected copy of a defective gene
What disorders are good candidates for gene therapy?
Genetic disorders caused by mutation in single genes.
What are vectors? What must they do?
The method used to get DNA into cells. They must target the right cells, integrate the gene in the cells, activate the gene, and avoid harmful side effects.
What are the advantages and disadvantages of using a viral vector?
Advantages: Good at targeting and entering cells. Some target specific types of cells. Can be modified so they can’t replicate and destroy cells.
Disadvantages: Carry limited genetic material, so some genes may be too big to fit into some viruses. Can case immune responses in patients, leading to sickness or the immune system blocking the virus from delivering the gene to cells or kill the cells once delivered.
Compare in vivo and ex vivo.
In vivo: Vector is directly injected into patient. Virus. More likely to trigger immune response. No verification before put into patient.
Ex vivo: Gene is delivered to cells removed from the body growing in culture. After gene is delivered, cells are put back in patient. Less likely to trigger immune response. Allows verification that cells are functioning properly before put in patient.
What are challenges of gene therapy?
Gene delivery and activation, immune response, disrupting important genes in target cells, ad commercial viability.
What are potential issues in gene delivery?
Activation issues and gene being incorporated into the wrong cells.
What is gene silencing?
Turning off a gene so no protein is made from it.
What is repairing mutations?
Replacing a defective copy of a gene with a working one.
What does genetically modifying immune cells to target specific molecules do?
Allows them to recognize a specific antigen and destroy its carrier cells.
What disorders have been treated with gene therapy?
Hereditary blindness, immune deficiencies, hemophilia, blood disorders, fat metabolism disorder, cancer, and Parkinson’s.
What are GMOs?
Genetically modified organisms whose DNA has been modified in the lab in order to favor the expression of a desired trait; an organism that has had a foreign gene inserted in its DNA.
What is a donor? What is a recipient?
Donor is the organism whose DNA is used for its desired trait (ex. bacteria). Recipient is the organism that will receive the donor’s DNA to carry out the process of protein synthesis with the desired trait.
What is rDNA? What does it do?
Recombinant DNA. It takes a piece of DNA, and combines it with another strand of DNA to create a new DNA strand.
What is a plasmid?
A circular piece of DNA floating free in a bacteria.
What do restriction enzymes do?
Cut an opening in the plasmid in order to insert the DNA, which is glued using ligase, and eventually copie through protein synthesis.