Flashcards in Molecular medicine II Deck (19):
What are the two prerequisites for Gene therapy?
1. Mutated gene that you are replacing must be recessive
2. The cells must be accessible to manipulation
What is somatic gene therapy?
Repairing somatic cells of the body
What is germ-line gene therapy?
Repairing genes in gametes
How are genes delivered for gene therapy?
What are the two main characteristics that are sought for a vector virus?
Aggressive spread (but not too much)
Effective integration into host DNA
What are the four, non-viral methods of gene delivery?
What are the advantages/disadvantages of using liposomes?
easy to use, commercially available
Slow, low integration
What are the advantages/disadvantages of using naked DNA?
easy to prep, can be large
DNA is quickly degraded
What are the advantages/disadvantages of using complexed DNA?
most stable than naked DNA, large
Not much more stable
What are the advantages/disadvantages of using artificial chromosome?
No insertion into genome required, no size limit
Unpredictable chromosomal events during meiosis
What are the steps of Ex vivio gene therapy?
1. Remove cells from pt
2. manipulate cells
3. reinsert back to pt
What is the function of adenosine deaminase? A lack of it lead to what? Why?
Break down deoxyadenosine
Immunodeficiency due to lymphocyts the toxic effects of dATP (Inhibits Ribonucleotide reductase)
What type of cells are best changed in in vivo gene therapy?
Epithelial cells or those readily accessible
What is the function of herceptin?
Antibody that binds teh HER@/Neu receptor for epidermal growth factor
What are the symptoms of Edward syndrome (4)?
What are the symptoms of Down syndrome (3)?
Low set ears
What are the symptoms of Patau sydrome(5)?
What are the symptoms of Turner syndrome (5)?
Carry angle decreased