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MOLBIO (LAB) > Gene Therapy and Gene Silencing > Flashcards

Gene Therapy and Gene Silencing Flashcards

1
Q

● Experimental technique that uses genes to treat or prevent disease

A

Gene therapy

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2
Q

● Currently being tested only for diseases that have no other cures.

A

Gene therapy

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3
Q

Enumerate the examples of diseases that have no other cures

A

Cancer, HIV, and other inherited diseases

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4
Q

● Designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein

A

Gene therapy

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5
Q

In gene therapy, you insert a normal gene to _________ or _________ for an abnormal gene

A

replace or compensate

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6
Q

DNA is transcribed to form mRNA which is translated to its product, protein (expressed product)

A

Centra dogma

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7
Q

Enumerate the approaches in gene therapy

A

Replacing mutated gene
Inactivating, or “knocking out”
Introducing a new gene into the body

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8
Q

The term used for the turn off the mutated gene

A

Inactivate

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9
Q

The term used to diminish the production or expression of the mutated gene

A

Knocking out

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10
Q

How does introducing a new gene into the body help?

A

Which will help fight against diseases

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11
Q

In which year does the genetic engineering was first presented at the 6th international Congress of Genetics

A

1932

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12
Q

In 1994, ________ and ________ suggested “genes could be transferred within nucleic acids”

A

MacLeod and McCarly

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13
Q

Concept of genetic correction arise

A

1944

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14
Q

In 1947, _________ was first to use the term gene therapy

A

Clyde E. Keeler

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15
Q

In which year: set the basis of molecular genetics and gene transfer

A

1960s and 1970s

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16
Q

In which year: The first deliberate transfer of foreign genes into human recipients, was performed by ____________

A

1970
Stanfield Rogers

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17
Q

Establishment of recombinant DNA technology

A

1973

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18
Q

In which year: Martin Cline at UCLA headed another highly controversial human trial designed to treat __________

A

July 1980
β-thalassemia

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19
Q

an inherited blood disorder in which the body doesn’t make normal hemoglobin.

A

β-thalassemia

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20
Q

There is a problem with the amino acid sequence of the β-globin chain (deletion)

A

β-thalassemia

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21
Q

In which year: Enzyme-producing-gene-corrected cells were further theorized as a viable approach for treating ________

A

1983
Lesch–Nyhan disease

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22
Q

x-linked recessive disorder

A

Lesch-Nyhan disease

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23
Q

T/F First serious approved gene therapy trials happened two decades later in the USA:

A

F; just another decade later

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24
Q

Advanced melanoma were treated with tumor-infiltrating lymphocytes isolated from solid tumors

A

First trial

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25
Q

Employed enzyme-transduced T-cells for adenosine deaminase (ADA) severe combined immunodeficiency

A

Second trial

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26
Q

What are the two types of gene therapy?

A

Somatic Cell Gene Therapy
Germ line cell gene therapy

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27
Q

Therapeutic genes transferred into somatic cells

A

Somatic Cell Gene Therapy

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28
Q

Example: introduction of genes into bone marrow cells, blood cells, skin cells etc

A

Somatic Cell Gene Therapy

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29
Q

T/F: In Somatic Cell Gene Therapy, it will be inherited in later generations

A

F; it will NOT be inherited

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30
Q

Somatic Gene Cell Therapy can be _____ or _______

A

Ex vivo
In vivo

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31
Q

Therapeutic genes transferred into germ cells (eggs and sperm cells)

A

Germ line cell gene therapy

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32
Q

Example: genes are introduced to egg cell or sperm cell

A

Germ line cell gene therapy

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33
Q

T/F: In germ line cell gene therapy. it will be inherited to next generation

A

T

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34
Q

● It only affects the targeted cells in the patient

A

Somatic cell gene therapy

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35
Q

● It cannot be transferred/passed to future generations (hindi namamana)

A

Somatic cell gene therapy

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36
Q

T/F: somatic cell gene therapy are short lived/short life-span

A

T

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37
Q

In somatic gene therapy, the transporting of gene to the target cell/tissue is _____________

A

problematic

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38
Q

Somatic gene therapy is acceptable/appropriate gene therapy for:

A

→ Cystic fibrosis
→ Muscular dystrophy
→ Cancer
→ Certain infectious diseases

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39
Q

● Has a possibility of inheritance

A

Germ line cell gene therapy

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40
Q

in germ line cell gene therapy, it has a potential of offering a ________ therapeutic effect to all those who inherit the target gene

A

permanent

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41
Q

T/F: in germ line cell gene therapy, it has the possibility of eliminating some diseases from a particular family.

A

T

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42
Q

● Raises controversies

A

Germ line cell gene therapy

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43
Q

Why are there controversies regarding germ line cell gene therapy?

A

→ There are concerns about the technical aspects of the experimental approach
→ Some people view this therapy as unnatural
→ Some of religious devotees likened it to playing with God

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44
Q

● Means external

A

Ex vivo gene therapy

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45
Q

→ Cells with the mutated genes will be modified outside the body and transplant it again in the body

A

Ex vivo gene therapy

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46
Q

In Ex vivo gene therapy:

Example: cells from patient’s ___________ or _________ are removed wherein they are isolated and cultured in the laboratory.

A

bone marrow or blood

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47
Q

T/F: in Ex vivo gene therapy, after the removal of cells from the patient, cells are then exposed to the virus that carries the desired gene (normal gene)

A

T

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48
Q

● Innovative

A

Ex vivo gene therapy

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49
Q

○ Proteins with genome within

A

Viruses

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50
Q

Basic pathogenesis of viruses

A

Infects healthy cells by inserting genome inside the cell

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51
Q

○ Virus used in ex-vivo is modified=_______ (________)

A

attenuated (non-infectious)

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52
Q

● Means Interior

A

In vivo gene therapy

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53
Q

→ Genes are changed in the cell within the body

A

In vivo gene therapy

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54
Q

T/F: in in vivo gene therapy, abnormal genes are changed after the cell is extracted from the patient

A

F: changed while the cell is inside the body

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55
Q

● The transfer of your corrected/normal/healthy gene requires a ________

A

Vector

56
Q

● Vehicle for gene delivery

A

Vector

57
Q

Vector

● Can be manipulated _______ to modify their genomes in order to insert a gene of interest

A

in vitro

58
Q

● 2 main class of vectors:

A

→ Viral vectors
→ Non-viral vectors

59
Q

IDEAL VECTORS

A

Target
Integrate
Activate
Avoid

60
Q

T/F: No universal vector exists

A

T

61
Q

● Cell with original genes

A

Viral vectors

62
Q

● Vector inserts a new gene into cell

A

Viral vectors

63
Q

New gene in the cell along with original genes

A

Viral vectors

64
Q

Types of Viral Vectors

A

Retrovirus
Adenovirus
Adeno-associated virus
Herpes simplex virus

65
Q

○ Majority of gene therapy clinical trial uses _________________

A

retrovirus & adenovirus

66
Q

What are the Adeno-associated virus

A

○ Lentivirus
○ Poxvirus
○ α-virus

67
Q

These viruses differ on how well they can transfer/insert the gene into the target cell.

A

Types of Viral Vectors

68
Q

● Involves the use of physical approach/carrier-free gene delivery (Naked DNA)

A

Non-Viral Vectors

69
Q

Non-Viral Vectors

Chemical Methods (uses synthetic vector)

A

→ Oligonucleotides
→ Lipoplexes and polyplexes

70
Q

Enumerate Non-viral methods

A

Naked DNA
Oligonucleotides

71
Q

Simplest method

A

Naked DNA

72
Q

Naked DNA

● _________ injection of a naked DNA plasmid

A

Intramuscular

73
Q

T/F: Injection of naked DNA intramuscularly has shown a very low expression in comparison of other methods.

A

T

74
Q

Naked DNA

Electroporation and the use of a “________”

A

gene gun

75
Q

uses high-pressured gas to shoot DNA-coated gold particles within the target cell.

A

Gene gun

76
Q

Inactivate the genes

A

Oligonucleotides

77
Q

● Uses antisense specific

A

Oligonucleotides

78
Q

Disrupt the transcription of the faulty gene = no production of mRNA

A

Antisense specific

79
Q

Oligonucleotides

● Uses small molecules of RNA called ______ (_____________)

A

siRNA (small interfering RNA)

80
Q

siRNA (small interfering RNA) is used to signal the cell to cleave specific unit sequences in the mRNA transcript of the faulty gene which will disrupt _______

A

Translation

81
Q

Oligonucleotides

Uses (single stranded/double stranded) oligodeoxynucleotides as a decoy for the transcription factors

A

double stranded

81
Q

Oligonucleotides

Uses (single stranded/double stranded) oligodeoxynucleotides as a decoy for the transcription factors

A

double stranded

82
Q

Chemical methods in Non-viral Vectors

A

Lipoplexes and polyplexes

83
Q

T/F: in lipoplexes and polyplexes, to improve the delivery of the new DNA within the cell, the DNA must be protected from damage and its entry into the cell must be facilitated.

A

T

84
Q

● New molecule have been created that have the ability to protect the DNA from undesirable degradation during the transfection process

A

Lipoplexes and polyplexes

85
Q

insertion of nucleic acid within eukaryotic cell

A

Transfection

86
Q

● Example of lipoplex is plasmid DNA can be covered with lipids.

A

Micelles
Liposomes

87
Q

When the organized structure is complexed with DNA it is called a ________

A

Lipoplex

88
Q

● The most common use of lipoplex is the gene transfer within a cancer cell wherein the supplied gene within the lipoplex has activated __________

A

tumor suppressor gene

89
Q

T/F: Tumor suppressor gene increases the activity of oncogenes

A

F; decreases

90
Q

Cancer genes

A

Oncogenes

91
Q

T/F: Recent studies show the lipoplex is useful in transfecting respiratory epithelial cells.

A

T

92
Q

three types of lipids

A

Anionic (negatively charged)
Neutral
Cationic (positively charged)

93
Q

Neutral

Anionic and neutral lipids were used for the construction of _______ for synthetic vectors.

A

Lipoplex

94
Q

T/F: Neutral lipids has a high amounts of toxicity but are also compatible with other body fluids

A

F; little toxicity

95
Q

○ They can adapt to become tissue specific

A

Neutral lipids

96
Q

○ Complicated and time-consuming to produce

A

Neutral lipids

97
Q

Naturally bind/form complex with negatively charged DNAs

A

Cationic (positively charged) lipids

98
Q

T/F: Since cationic lipids are positively charged, they can interact well with the cell membrane.

A

T

99
Q

■ The insertion of lipoplex is easier from within the cell through the process of ________

A

endocytosis

100
Q

○ The cationic property or cationic lipids can protect DNA against _______

A

degregation

101
Q

What is another form of lipids

A

Polyplexes

102
Q

→ A complex of polymers with DNA

A

Polyplexes

103
Q

Polyplexes

→ Most consist of (ionic/cationic) polymers and their production are regulated by ionic interaction

A

cationic

103
Q

Polyplexes

→ Most consist of (ionic/cationic) polymers and their production are regulated by ionic interaction

A

cationic

104
Q

→ One large difference between the method of action of lipoplexes and polyplexes is that polyplexes (can/cannot) release their DNA load into the cytoplasm

A

cannot
cytoplasm

105
Q

Coat transfection with endosome lipid agent such as _______________ must occur.

A

inactivated adenovirus

106
Q

● Is a technique that aims to reduce or eliminate the production of a protein from it corresponding gene

A

Gene silencing

107
Q

Gene silencing is described as “_______” of a gene by a mechanism other than genetic modification

A

Switching off

108
Q

● Occurs when RNA is unable to make a protein during translation

A

Gene silencing

109
Q

T/F: Gene silencing is completely similar from gene knockout

A

F; completely different

110
Q

the expression of the gene is reduced

A

Gene knockdown

111
Q

gene is completely erased from the organism’s genome

A

Gene knockout

112
Q

ADVANTAGES OF GENE SILENCING

A
  1. It is cost-effective
  2. It can induce viral resistance
  3. It is a powerful tool for analyzing unknown genes in a sequence genome
  4. Useful approach in future gene therapy
113
Q

HOW DOES GENE SILENCING WORK?

Accomplished by binding a specific strand of RNA to an existing _______ strand

A

mRNA

114
Q

example of specific strand of RNA binded during gene silencing?

A

siRNA (it will inhibit translation)

115
Q

TYPES OF GENE SILENCING

A

Transcriptional gene silencing
Post transcriptional gene silencing

116
Q

Promoter silenced

A

Transcriptional gene silencing

117
Q

Transcriptional gene silencing

Genes hypermethylated in ___________ region

A

promoter

118
Q

Purpose of transcriptional gene therapy

A

Viral immunity

119
Q

Promoter active

A

Post transcriptional gene silencing

120
Q

Post transcriptional gene silencing

Genes hypermethylated in _________ region

A

Coding

121
Q

Purpose of post transcriptional gene silencing

A

Viral immunity

122
Q

● A result of histone modification

A

Transcriptional gene silencing

123
Q

→ When histones are modified, it will make it inaccessible to transcriptional machinery such as:

A

○ RNA polymerase
○ Transcription factors

124
Q

● Formation of mRNA

A

Post transcriptional gene silencing

125
Q

T/F: The formation of mRNA suppress the expression of mRNA (translation)

A

T

126
Q

● Deepen the pigmentation in petunia

A

1990 Jorgensen

127
Q

Introduction of transgenes homologous to endogenous

A

1990 Jorgensen

128
Q

the introduction of transgenes homologous to endogenous in 1990 jorgensen often resulted in plants with both genes suppressed called ______

A

co-suppresion

129
Q

Degradation of the endogenous and transgene.

A

1990 Jorgenses

130
Q

Injection of either antisense or sense RNAs in the germline of ________

A

1995 Guo and Kemphues
Candida elegans

131
Q

The 1998 Craig Mello and Andrew Fire is an extended experiment of _______, ____, and _______

A

Jorgensen, Guo, and Kemphues.

132
Q

● Combination of sense and antisense

A

1998 Craig Mello and Andrew Fire

133
Q

● RNA(=dsRNA).

A

1998 Craig Mello and Andrew Fire

134
Q

T/F: Double-stranded RNA is 2x more effective than single stranded RNA

A

F; 10x

MOLBIO (LAB) (3 decks)

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