Development Disruptions
Cleavage stops early. What structure fails to form?
These cells from the inner cell mass of a blastocyst can give rise to almost all embryo cell types but NOT the placenta.
What are embryonic stem cells?
Two repair pathways after CRISPR cuts DNA
What are NHEJ and HDR?
Treating a disease by altering a patient’s DNA is known as this.
What is gene therapy?
Inner cell mass is destroyed. What is the outcome?
What is no embryo develops?
iPSCs are created using what type of mechanism?
What is activation of pluripotency genes (Yamanaka factors)
This RNA molecule in CRISPR directs Cas9 to the correct DNA target — and can be custom-designed by researchers to target any gene.
What is guide RNA?
A gene edit made to a patient's blood cells to treat sickle cell disease — this type affects only the individual and is NOT heritable.
What is somatic gene editing?
If all cells have identical DNA, what explains why a neuron and muscle cell are different?
What is differential gene expression?
Major risk of stem cell therapy?
What is tumor formation?
A researcher wants to fix the single-base mutation causing sickle cell disease. The two things they must provide beyond the Cas9 protein itself.
What are (1) a gRNA targeting the mutation site and (2) a healthy DNA repair template for HDR?
Doctors generate stem cells from a patient’s own skin cells and use them for therapy. What major clinical advantage does this provide over donor cells?
What is reduced immune rejection?
Cells express ALL genes equally. Predict outcome.
What is no specialization
What are the two main regeneration strategies.
What are stem cells and dedifferentiation"
To fix a mutation precisely using CRISPR/Cas9, what must you provide?
What is a repair donor DNA template?
In 1982, the scientific community accepted genetically engineered insulin because it modified this organism
What is bacteria (E. coli)?
Signaling gradients are lost. What fails at organism level?
A cell is reprogrammed but fails to differentiate later. What went wrong?
What is lack of proper signaling cues
To knock out a gene entirely using CRISPR, you design a gRNA targeting that gene and rely on this repair pathway, which introduces random mutations.
What is NHEJ?
He Jiankui's 2018 experiment was condemned because he performed this type of gene editing in humans: the CCR5 mutations would be passed to all future descendants.
What is germline editing?