Use LEFT and RIGHT arrow keys to navigate between flashcards;
Use UP and DOWN arrow keys to flip the card;
H to show hint;
A reads text to speech;
18 Cards in this Set
- Front
- Back
|
Gene therapy |
a novel approach to treating disease based on modifying the expression of a person's genes toward a therapeutic goal |
|
|
somatic gene therapy |
manipulation of gene expression in cells so as to be corrective for the patient |
|
|
germline gene therapy |
involves genetic modification of germ cells that will pass the selected change onto the next generation |
|
|
What do the vast majority of clinical trials involving gene therapy treat? |
cancer |
|
|
are there any FDA approved gene therapy products currently on the market?? |
Nah |
|
|
therapeutic strategies of gene therapy |
vector carries a gene that encodes a protein that is either defective or not present due to mutations in the patients' endogenous genes |
|
|
cytolytic strategy of gene therapy |
vector is designed to destroy or eliminate a diseased cell or tissue (often induces production of toxic product) |
|
|
viral vectors |
gene delivery vehicles trick is to remove disease-causing components and insert recombinant genes that will be therapeutic |
|
|
liposome transfection |
complexes of DNA and lipids |
|
|
"Gene Gun" technology |
delivery of DNA on gold particles, primarily for DNA vaccines |
|
|
adenovirus |
episomal high transduction efficiency infects replicating AND non-replicating elicits immune response |
|
|
adeno-associated virus |
integrates into human chromosome 19 low immunogenicity no associated disease infects replicating AND non-replicating |
|
|
herpes |
large insert capacity broad host range replicating AND non-repicating cells |
|
|
liposomes/naked DNA |
no limit to size of genes that can be delivered low immunogenicity poor levels of gene transfer |
|
|
retrovirus |
non-pathogenic transduces only NON-DIVIDING cells long term expression inactivated by human complement |
|
|
Which vectors infect both replicating and non-replicating cells |
Adenovirus adeno-associated virus herpesvirus |
|
|
Which vector is best for getting into non-dividing cells? |
HIV/Lentivirus/Retrovirus vectors |
|
|
CRISPR/Cas9 system |
allows for specific gene editing by expressing Cas9 and specifically designed cRISPRs such that any organism's genome can be cut at any desired location allowing specific replacement of a defective gene with a functional one |
