The first Tedx Talk
Data I barely even know Ha
That means something... Right?
What's wrong?
Review
100
A reference SNP for a disease
Tag SNP
100
Kaplan meier plot
100
how long after administration does the therapeutic exert a measurable effect
Biodistribution
100
Modifier Gene for SMN1 mutation
SMN2
100
A receptor tyrosine kinase that activates KRAS
EGFR
200
An ASO treatment for Spinal Muscular Atrophy
Spinraza
200
Explain a swim plot in terms of Casgevy
Each row is a person, they are treated and tracked (Hospitalizations for casgevy) depending on the disease
200
the pattern of distribution of a substance, often a therapeutic agent or drug, within a living organism after administration
Pharmacodynamics
200
●a research method that compares the genomes of many people to find genetic variations associated with specific diseases or traits
GWAS (genome wide association study)
200
GLP1 binds to this type of receptor
GPCR
300
Worst Isoform of ApoE
4
300
Improves stability and prevents degradation of ASO
2-MOE
300
how does the human body interact with the therapeutic after its administration
Pharmacokinetics
300
Explain the ASO Mechanism of Action
Rnase H
300
Explain siRNA mediated degradation
DSRNA, DICER, RISC Complex
400
The date of your next midterm
10/29
400
Explain a microwalk assay and how it pertains to SMA
ASOs "walked" across this region of the RNA to identify the positional effects of specific nucleotides on the splicing of exon 7. The purpose was to precisely map which intronic positions influence exon 7 inclusion or skipping, as skipping of exon 7 in SMN2 leads to reduced functional SMN protein and causes SMA.
400
a predefined outcome or event that is used to objectively measure the efficacy or safety of the therapeutic under investigation
Endpoint
400
regulates the splicing of the SMN gene's transcript, influencing exon 7 inclusion in the SMN protein
hnRNP
400
Make an antibody name 2 ways to make a humanized antibody
Transgenic mouse, phage display
500
Explain the mechanism of CRISPR
Guiderna and TRACRrna, cas9, cut
500
Used to measure a child's motor function
Hine-2 Score
500
Does Response Curve
relationship between amount of therapeutic and some disease-relevant outcome
500
UH OH you have too much EGFR and it is still activating what's happening?
ligand independent dimerization
500
in T-cells, keeping
them in an inactive
state to limit possible
autoimmune
response, but also
limiting anti-tumor
response
CTLA4