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Brain Disorders > Gene therapy for Parkinsons > Flashcards

Gene therapy for Parkinsons Flashcards

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1
Q

What is gene therapy?

A

Use of bacterial virus to transport genetic material into cells/tissues

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2
Q

What is the aim of gene therapy?

A

Halt the process of neurodegeneration in inherited neurological disorders

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3
Q

What does gene therapy treat?

A

Gene therapy will affect motor symptoms & target other symptoms due to dysfunction of brain circuits eg speech

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4
Q

What is DNA-mediated gene rediting represented by?

A

CRISPR-cas9

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5
Q

What 3 things does RNA-mediated protein silencing involve?

A

siRNA
shRNA + dicer
antisense oligonucleotides

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6
Q

What is CRISPR?

A

Technique that allows precise alterations to be made in genomic DNA sequences by a process known as genome editing

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7
Q

What does CRISPR give bacteria?

A

ability to recognize precise genetic sequences

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8
Q

What are the enzymes associated with CRISPR called?

A

Cas

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9
Q

What is MoA of Cas9?

A

cut target sequence

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10
Q

What does the synthetic dsRNA contain?

A

Complementary sequence and targets place where there is a mutation

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11
Q

Why is non-homologous end joining bad?

A

Prone to errors
Could potentially disrupt gene function

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12
Q

Why is HDR better?

A

Template provided with specific change in DNA sequence
Change will be inserted in genome
Repair double stranded break
New experimental DNA with sequence we want

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13
Q

Describe the CRISPR + Cas9 genome diting technique?

A

RNAs direct the nuclease Cas9 to selected sequences of genomic DNA

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14
Q

What is Cas9?

A

Molecular scissors recruited to cut both strands at a precise location & repair or remove mutation

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15
Q

What is a benefit of CRISPR?

A

Repair any mutation at any location in genomic DNA

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16
Q

What is used as vector to reach nuceli?

A

Adeno-associated virus

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17
Q

What are 2 types of delivery of CRISPR Cas9?

A

Viral eg. adenovirus
Non-viral eg. nanoparticles

18
Q

What 3 things does muscle-restricted Cas9 expression enable?

A

Editing of mutation
Multiexon deletion
Complete gene correction via homologous recombination

19
Q

How is CRISPR-Cas9 used to treat PD?

A

Elicit down regulation of erroneous SNCA gene expression levels

20
Q

What did CRISPR-Cas9 restore in PD?

A

Physiological levels of SNCA mRNA -> allowed dopaminergic neurons to maintain function

21
Q

What are 3 challenges of CRISPR-Cas9?

A

Non-specific editing of DNA in other locations
Accidental changes that can be permanent
Unwanted immune response

22
Q

What 2 things does Cas-9 trigger in the immune response?

A

Humoral response
Specific antigen T-cells

23
Q

Why is RNA gene therapy better than DNA gene editing?

A

Only temporary regulations of gene expression

24
Q

In RNA therapy what are 2 options if there are accidental adverse effects?

A

Discontinuation possible
Rapid degradation of RNA nucleotides

25
Q

What is the MoA of dsRNA?

A

dsRNA -> transfect cells -> binds to complimentary mRNA -> protein depletion

26
Q

What is the MoA of siRNA?

A

RNA molecules inhibit gene expression or translation by neutralizing mRNA molecules

27
Q

What complex is siRNA incorporated in?

A

RISC

28
Q

What can siRNA gene therapy be used to treat?

A

AD

29
Q

What are 4 challenges of siRNA?

A

unwanted immune response
poor diffusion within brain tissue
requires injections of liposomes or polymers
delivery procedures & stable expression

30
Q

Why are nanoparticles used?

A

carriers for siRNAs with controlled delivery efficiency & low toxicity profiles

31
Q

How are shRNAs delivered?

A

AAV

32
Q

How is shRNA different to siRNA?

A

Integrated into host genome - continually expressed for months
Uses endogenous processing that exists in the cell

33
Q

What are 3 challenges of shRNA?

A

Risk of overdose due to excessively strong promoters
clog up miRNA
shRNA-mediated toxicity

34
Q

What are antisense oligonucleotides?

A

single stranded nucleotides that complementary bind mRNA to suppress mutant protein expression

35
Q

How long is mutant protein expression suppressed for after ASO therapy?

A

12 weeks

36
Q

What is an advantage of ASO?

A

Good diffusion in brain
absorbed efficiently by cells

37
Q

How is ASO administered?

A

Lumbar injection

38
Q

What 3 things does ASO suppress in PD?

A

LRRK2
alpha syn synthesis
amelioration of motor symptoms

39
Q

What is LRRK2 linked to?

A

alphasyn synthesis

40
Q

What are 3 benefits of ASO?

A

No strong immunological response
low cost
negligible side effects

41
Q

What 2 things does ASO gene therapy require?

A

evaluation of degree of disease modifying effect
and long term efficiency

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