CRISPR Basics:
What molecule does embryonic genome editing change?
DNA
Discovery of CRISPR
(1987-2012)
What molecule recognizes the target DNA?
Guide RNA
Which repair method stands for NHEJ?
Non-homologous end joining
Name one disease listed that genome editing could potentially help prevent
Cystic fibrosis, Huntington’s, OR Tay-Sachs
Embryonic genome editing changes DNA at what stage of development?
Early embryo
In what year did CRISPR enter human cells?
2013
What protein cuts the target DNA?
Cas9
Which repair pathway does NOT require a template?
NHEJ
What term describes accidental mutations occurring somewhere other than the intended target?
Off-target mutations
More than what percentage of rare diseases have a genetic cause?
80%
In what year did the first nonviable human embryo editing occur?
2015
What are the three general steps of CRISPR/Cas9 genome editing?
Recognition, cleavage, repair
Which repair pathway is precise but less efficient?
HDR
What term refers to concerns about genetically selecting or enhancing desired traits?
Eugenetics or ”designer babies”
What three types of DNA changes can scientists make during repair according to the slides?
Deletion, modification, or addition
What controversial genome-editing case occurred in 2018?
The He Jainkui Case
During recognition, what does guide RNA do?
Recognizes the target DNA and directs Cas9 to it
During which two phases of the cell cycle can HDR occur?
S and G2 phases
Name 2 bioethical concerns listed in the presentation
Informed consent, human experimentation, eugenics/designer babies, unpredictable long-term health issues
Why could editing embryos potentially be useful for families with inherited genetic risks?
It could help prevent/eliminate heritable disease
We mentioned base editing in connection with what syndrome?
Marfan Syndrome
After Cas9 cuts the DNA, what three outcomes can scientists produce during the repair stage?
Deletion, modification, or addition
Which DNA repair pathway is more precise but less efficient, requires a donor DNA template, and can be used to correct mutations or insert genes?
Homology-directed repair (HDR)
According to the final “Now What?” list, what 2 things are needed moving forward?
More safe/comprehensive research + attention to legal and ethical guidelines