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Enzymes > Molecular medicine II > Flashcards

Molecular medicine II Flashcards

1
Q

What are the two prerequisites for Gene therapy?

A
  1. Mutated gene that you are replacing must be recessive

2. The cells must be accessible to manipulation

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2
Q

What is somatic gene therapy?

A

Repairing somatic cells of the body

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3
Q

What is germ-line gene therapy?

A

Repairing genes in gametes

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4
Q

How are genes delivered for gene therapy?

A

Viral/non-viral vector

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5
Q

What are the two main characteristics that are sought for a vector virus?

A 
Aggressive spread (but not too much)
Effective integration into host DNA
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6
Q

What are the four, non-viral methods of gene delivery?

A

Liposomes
Naked DNA
Complexed DNA
Artificial chromosome

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7
Q

What are the advantages/disadvantages of using liposomes?

A

easy to use, commercially available

Slow, low integration

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8
Q

What are the advantages/disadvantages of using naked DNA?

A

easy to prep, can be large

DNA is quickly degraded

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9
Q

What are the advantages/disadvantages of using complexed DNA?

A

most stable than naked DNA, large

Not much more stable

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10
Q

What are the advantages/disadvantages of using artificial chromosome?

A

No insertion into genome required, no size limit

Unpredictable chromosomal events during meiosis

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11
Q

What are the steps of Ex vivio gene therapy?

A
  1. Remove cells from pt
  2. manipulate cells
  3. reinsert back to pt
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12
Q

What is the function of adenosine deaminase? A lack of it lead to what? Why?

A

Break down deoxyadenosine

Immunodeficiency due to lymphocyts the toxic effects of dATP (Inhibits Ribonucleotide reductase)

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13
Q

What type of cells are best changed in in vivo gene therapy?

A

Epithelial cells or those readily accessible

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14
Q

What is the function of herceptin?

A

Antibody that binds teh HER@/Neu receptor for epidermal growth factor

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15
Q

What are the symptoms of Edward syndrome (4)?

A

rocker feet
Prominent occiput
Synpolydactyly
Heart defects

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16
Q

What are the symptoms of Down syndrome (3)?

A

Low set ears
Wide eyes
Heart defects

17
Q

What are the symptoms of Patau sydrome(5)?

A 
Cutia aplasia
Midline defects
Holoprosencephaly
Heart defects
club feet
18
Q

What are the symptoms of Turner syndrome (5)?

A 
Small stature
Skin fold
Carry angle decreased
Amenorrhea
Gonadal dysplasia
19
Q

What are the symptoms of Klinefelter syndrome (5)?

A 
Sparse facia hair
behavior issues
Small testes
DM
cynecomasita

Enzymes (24 decks)

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