General Info
What is the main symptom of HTT?
Chorea
What is neuropathology?
The study of diseases of the nervous system.
What is the mutant HD gene?
mHTT
What does ameliorate mean?
To reduce/to improve.
Which motor test had opposite results than expected?
Balance beam
What is a polyQ chain?
A strand of CAG repeats.
What brain region is degeneration primarily seen in HD?
Striatum, MSN, hippocampus
What are the two guide RNAs being used?
T1 and T3
What does CRISPR stand for?
What does KI (knock-in) mean for the mouse model?
Knock-in refers to placing an exogenous (human model) gene into the mouse model at a selected loci.
What is CRISPR/Cas9?
Cas9 is the cutting protein, CRISPR is what is attached to the edited DNA.
What is the abbreviation for the HD mutant mouse?
HD-140Q-KI
How many repeats of polyQ results in full penetrants of HD?
40+
What were the antibodies used in the immunohistochemical studies?
GFAP and NeuN antibodies
What is the name of the virus that delivered the gRNA?
AAV-HTT-gRNA