Therapeutic Approaches
HARD LEVEL (Who was paying attention 👀) Cell Replacement Therapy
Characterization
HD in the Whole Body
100

What are 5 imaging methods for HD?

MRI, fMRI, DTI, MRS, PET

100

Are FNPs pluripotent or multipotent?

multipotent

100

HD is characterized by the loss of what in which brain region?

MSNs in striatum

100

What over-the-counter medication could possibly reduce the risk of development of Alzheimer's and Parkinson's? 

Ibuprofen

200

What three approaches are the most promising?

Antisense Oligonucleotides (ASOs), RNA interference (RNAi), & Zinc Fingerprinting

200

What 3 issues do primary fetal cells present?

Scarce, difficult to standardize, cannot be stored long-term

200

What motor movements are affected by HD? What are the symptoms/names for these?

Involuntary - chorea, Voluntary - bradykinesia
200

What is the main cause of death in HD patients? 

Unknown 

or 

Possibly cancer

300

What are two mice models for Huntington's Disease?

Conditional knockout model and Nucleotide based gene silencing

300

hFNP grafts lack organization in what zones?

P-zones and non-P zones

300

What is the CAP equation?

AGE × (CAG – L)

300

What peripheral metabolic pathway is affected in Huntington's disease? 

Tryptophan Metabolism 

400

What is a potential risk for using gene silencing as a therapeutic approach? (more than one correct answer)

Lowering the WT HTT gene

Toxicity or neuroinflammation from synthetic oligonucleotides

Immunogenicity when using AAV-delivered compounds 

400

What is the peak period of MSN neurogenesis in humans?

8-10 weeks gestation

400

What cures Huntingtons disease?

Trick Question! There's still no cure, but we're getting close!

400

Sleep phenotypes associated with mHTT are not socially mediated between cohabiting mutation carriers

true or false 

false

500

Biomarkers related to what are likely to be important in the future?

Neuronal cell death and neuronal cell dysfunction

500

Name the 3 main pluripotency markers?

alkaline phosphatase (AP), Oct-4, SSEA-1

500

What disorder is caused by a mutation in chromosome 4?

Huntington's Disease!

500

What experimental immune modulator drug is currently under investigation in multiple sclerosis trials? 

Laquinimod 

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