Unit 2 Lesson 2 Flashcards
Gene Therapy, CRISPR cas9, Reproductive Tech
What is the overall goal of gene therapy?
To treat or get rid of genetic disorders/diseases by mutating the gene responsible.
What is the role of Vectors in gene therapy?
Vectors are used to transport the functional gene to the target cell and mutate the dysfunctional gene
What is the most common vector used in gene therapy?
Adeno- associated virus
How many base pairs can an Adeno-associated virus hold, what cells can it target, will it trigger an immune response, Will it integrate into the host cell genome, and is it efficient?
5000; a variety; Not likely; yes; Very
An Adeno-Associated virus needs a ______ ______ to replicate
Helper Virus
What are the considerations to make before choosing the correct vector to use during gene therapy?
The amount of base pairs it holds, whether or not it will trigger an immune response, whether or not it will integrate into the host cell genome, and the cells it can target (cell specificity )
What are the three strategies for in gene modification?
Insertion, Disability, and Reparation
What is the purpose of Insertion?
To give the functional gene to the patient
What is the purpose of Disability?
To disable the dysfunctional gene
What is the purpose of Reparation?
To repair the dysfunctional gene so it produces the functional protein.
What does CRISPR stand for? What does cas9 stand for?
CRISPR: Clustered Regularly Interspaced Short Palindromic Repeats
cas9: CRISPR associated
What does the CRISPR system do overall?
Guides RNA that tells system where to cut the gene.
Where does CRISPR take place?
Inside a nucleus.
CRISPR cas9 complex _____ on to a short ________ of DNA based on the ________ identified in the system.
locks; segment; sequence
cas9 ______ DNA and matches it to ___
Unzips; RNA
When completed, DNA is ___ and the gene is _________
cut; inactivated
The ____ ____ DNA will replace the inactivated gene
host cell
New _________ gene is added to the ___ ____
Functional; cut ends
What two benefits of CRISPR?
- Can be done without a vector
- Can target multiple genes at a time
What can CRISPR be used in?
basic research, agriculture, drug development, and treating patients with genetic disease
What is the role of guide RNA in the CRISPR process?
to show the cas9 where the restriction sites are located.
What diseases have been successfully treated using gene therapy?
Hemophilia, certain types of Cancer, HIV, etc.
What does a helper virus do?
It allows the vector virus to replicate
In vivo vs. Ex vivo
Inside the body, outside of the body
