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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 ________

56
Q

● Vehicle for gene delivery

57
Q

Vector

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

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

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

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.

74
Q

Naked DNA

Electroporation and the use of a “________”

75
Q

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

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.

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 ________

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

91
Q

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

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.

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.

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

103
Q

Polyplexes

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

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

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

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

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)

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

MOLBIO (LAB) (3 decks)

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