Theraputic Approaches to NeuroD. Disease

Question 1

Characteristics on NDD

Answer 1

Abnormal protein accumulation

Peferential death of certain neurons

Question 2

Huntington's Disease
Symptoms
Pathophysiology
- Lost Cells:
- Expansion
Treatment

Answer 2

Symptoms

• Chorea
• Altered basal ganglia

Pathophysiology

• Lost Cells: Medium spiny neurons in caudate & putamen
• Expansion of CAG
• Mutant huntingdon protein (toxic gain of function)

Treatment
- Tetrabenzadene = chrorea control

Question 3

Alzheimer’s Disease

Symptoms

Answer 3

Failure in at least one of the following cognitive functions

• Language skills
• Visual spatial orientatation
• Spatial orientation
• Abstract thinking
• Judgement
• Cortical shrinkage

Question 4

Alzheimer’s Disease

• Which cells lost?
• What accumulates?

Answer 4

• Neurons sensitive to neuron growth factor in the cortex, hippocampus, amygdala & cholinnergic basal forebrain neurons
• Loss of Ach
• Neurofibrillary Tangle and Amlyoid

Question 5

What is in Neurofibrillay tangles?

Answer 5

• Tau protien that use to hold microtubles togethether

Question 6

What is in Amyloid plaques?

Answer 6

• Amyloid Precursor Proteins cleaved by beta & gama secretase creates AB40/42

Question 7

In what molecular structure is Gamma secretase located

Answer 7

• Lipid rafts

Question 8

What physiological effect do amyloid plaques have?

Answer 8

• Altered axonal transport
• Altered synaptic activty
  Alterd Ca2+ concentrations

Question 9

Treatment options of Alzheimer’s Disease?

Answer 9

• Inhibit Acherase
• Small molecule inhibitors against amyloid plaques
• Statins to control choleestrol since gamma secretase is in a lipid raft

Question 10

Syptoms of Parkinson’s Disease

Answer 10

• COnstant tremor
• Less spontanous movement
• Limb rigity

Question 11

Parkinson’s Disease Pathophysiology

• Lose which cells
• What accumulates
• What is mutated

Answer 11

• Loss of dopaminergic neurons in substantia nigra
• Lewy Bodies accumulate
• Parkin & OCHL-1

Question 12

What is in Lewy Bodies?

Answer 12

• alpha-synuclein * ubiquit

- failure to degrade proteinss

Question 13

What is the role of Parkin & OCHL-1

Answer 13

Proteins involved in delivery of proteins to the proteosome

Question 14

3 ways to prevent disease progression

Answer 14

gamma-secretase inhibitors
small molecule inhibitos
Abs directed at amyloid

Question 15

3 ways to replace NT and which NT do you replaces

Answer 15

Implant dopamine secrting cell
Inhibit dopamine break down
Oral L-dopa & carbidopa

Question 16

Why is L-dopa given with carbidopa

Answer 16

Carbidopa diven to relieve symptoms of excess dopamine & prevents peripheral dopamine causing side-effects

Question 17

2 mechanisms to alter neuronal circutts

Answer 17

Partial palllidectomy

Deep brain stimulation

Question 18

Why is a partial pallidotomy effective?

Answer 18

Reduce over activity of the globus pallidus

Question 19

Where is DBS targeted

Answer 19

Subthalamic nucleus

Question 20

Name 2 viruses used in attempt for gene therapy

Answer 20

Adenovirus & retrovirus

Question 21

Name 2 neurotrophic factors that can be used to slow neuron loss with neurotrophic factors

Answer 21

• Neurotrophin

* Glial derived neurotrophic factor

Question 22

Name 3 items that can be transplanted

Answer 22

1. Embryonic dopamine producing cells
2. Autografts, xenograft and gentically modified cells
3. Stem cells