The Central Dogma
Where does translation take place?
The Ribosome
What are the two components of the CRISPR cas 9 complex?
Cas 9 protein, Guide RNA
What is NHEJ?
Non-homologous End Joining
Name two kinds of genetic medicines or techniques
Gene therapy, CRISPR cas 9, Exon skipping, RNA interference, small molecule drugs, gene switches
In what direction is DNA synthesized?
5' to 3'
True or false: nucleotides with Uracil nitrogenous bases usually have an OH group on the C2' of their sugar.
True
What is the region between CRISPR sequences, corresponding to genes of foreign bodies called?
Spacers
What does HDR stand for?
Homology directed repair
What form of genetic medicine silences regions of genes by affecting its ability to be spliced into the mature mRNA
Exon Skipping
What are the two most common ways small molecule drugs can interact / affect proteins?
Inhibition / promotion of activity
What enzyme unzips DNA during replication?
Helicase
Why is Cas9 referred to as an endonuclease?
It is capable of cutting nucleic acids
What does PAM stand for?
Protospacer Adjacent Motif
What are the two kinds of vectors used in gene therapy?
Integrating vectors and non-integrating vectors
What does Cas stand for?
Crispr Associated Protein
What process involving the removal of introns leads to the production of mature mRNA?
RNA splicing
What three components act on the pre-crRNA to create the CRISPR complex? Either the proteins or their function is an acceptable answer
Cas (9), tracr RNA, RNase III
List the 4 (or 5) steps involved in CRISPR Cas 9 gene editing
Targeting and recognition, binding to the protospacer, cleavage, DNA repair
Scientist sometimes use gene switches in order to reprogram the promoter region of a gene. This can be programmed to be turned on or off indefinitely, but also respond to certain stimuli. What might be an advantage of linking the cells expression to certain stimuli as opposed to causing it to be constrictively active or inactive?
Reversibility, allows us to test the activity of a particular gene directly
How is CRISPR delivered in vivo?
Using Gene therapy, the code for the cas 9 protein and guide rnas are supplied through a viral vector
Fill in the blank: During Transcription, RNA Polymerase advances ______’ -> ____’ on the ______template strand melting duplex dna and adding rNTPs to growing RNA
3', 5', lower
What does CRISPR stand for?
Clustered, Repetitive, Interspaced, Short, Palindromic Repeats
Why is the HDR mechanism typically less error prone than the NHEJ method? Bonus(What is the limitation of HDR?)
HDR uses a template strand, which ensures correct sequence repair more often
What kind of virus vectors are commonly used for gene therapy techniques leveraging non integrating vectors (acronym is fine)
AAV (adeno - associated viruses)
What is one of the limitations associated with the use of CRISPR Cas 9 in genetic medicine? (One point for each right answer)
Off-Target Effects, Protospacer Adjacent Motif Requirement, DNA-Damage Toxicity, Immunotoxicity, Precision Gene Editing With CRISPR, Delivery of CRISPR Gene Therapy