19.2.4

Gene Therapy

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Gene Therapy

Gene therapy is a genetic engineering technique used to cure disease.

Procedure

Procedure

  • Gene therapy involves the introduction of a target gene (the gene that confers a beneficial trait) into the genome.
    • The genome has been transformed.
  • The target gene is then transcribed and translated to produce the desired protein.
  • The protein counteracts the effect of a disease that is caused by a mutation.
Allele interactions

Allele interactions

  • Gene therapy is used to treat diseases that are caused by a mutation in a gene.
  • The way that gene therapy is used depends on the allele interactions of the gene that causes the disease.
    • If the mutation is in the recessive allele, a wild-type (typical of the species) dominant allele is inserted into the genome. The dominant allele counteracts the mutant alleles.
    • If the mutation is in the dominant allele, an allele that 'silences' the mutant allele is inserted in the genome.
Vectors

Vectors

  • Gene therapy uses vectors to insert the target gene into the genome.
  • Vectors transport allow the gene to be taken up by the cells of the host. The genome is then transformed.
    • Types of vector include plasmids, bacteriophages, liposomes, & naked DNA.
  • When choosing a vector, you need to consider factors such as the target cell, size of the gene to be inserted, and any immune response that may be caused.
    • For example, bacteriophages are a type of virus so may cause an immune response. They may, however, be able to deliver larger genes than naked DNA. Naked DNA is sensitive and easily broken down.
Types of gene therapy

Types of gene therapy

  • There are two types of gene therapy -
    • Somatic therapy - altering of alleles in adult body cells. This is a short term therapy and needs repeating.
    • Germline therapy - altering of alleles in sex cells. This is illegal in humans.
Example - cystic fibrosis

Example - cystic fibrosis

  • Cystic fibrosis is caused by a mutation in the CFTR gene.
  • The CFTR gene codes for a chloride ion channel that or transports Cl- ions out of cells and into mucus.
  • Cl- ions lower the water potential of mucus. This causes water to leaves cells and enter mucus by osmosis.
  • So, when the channel is mutated Cl- ions do not enter mucus. Mucus becomes thick & sticky.
    • This leads to problems such as a build-up of mucus in the lungs, blockage of digestive system ducts, and clogging of the cervical canal with mucus.
Cystic fibrosis - gene therapy

Cystic fibrosis - gene therapy

  • Treating cystic fibrosis with gene therapy would involve delivering a correctly function CFTR protein to affected cells.
  • This therapy is the subject of multiple clinical trials and has shown some promise.
Jump to other topics
1

Cell Structure

1.1

Cell Structure

1.1.1Studying Cells - Microscopes1.1.2Introduction to Eukaryotic & Prokaryotic Cells1.1.3Ultrastructure of Eukaryotic Cells1.1.4Ultrastructure of Eukaryotic Cells 21.1.5Ultrastructure of Eukaryotic Cells 31.1.6Prokaryotic Cells1.1.7Viruses1.1.8End of Topic Test - Cell Structure1.1.9Exam-Style Question - Microscopes1.1.10A-A* (AO2/3) - Cell Structure
2

Biological Molecules

2.1

Testing for Biological Modules

2.1.1Tests for Sugars & Starch2.1.2Tests for Lipids & Proteins
2.2

Carbohydrates & Lipids

2.2.1Monomers & Polymers2.2.2Carbohydrates2.2.3Examples of Carbohydrates2.2.4Triglycerides & Phospholipids2.2.5Types of Fatty Acids2.2.6Exam-Style Question - Carbohydrates2.2.7End of Topic Test - Carbohydrates & Lipids
2.3

Proteins

2.3.1Amino Acids & Overview of Protein Structure2.3.2Primary & Secondary Structure2.3.3Tertiary & Quaternary Structure2.3.4Globular & Fibrous Proteins2.3.5Haemoglobin2.3.6Collagen2.3.7End of Topic Test - Proteins
2.4

Water

2.4.1Water Structure & Function2.4.2End of Topic Test - Water
3

Enzymes

3.1

Enzymes

3.1.1Enzyme Action3.1.2Factors Affecting Enzyme Activity3.1.3Factors Affecting Enzyme Activity 23.1.4A-A* (AO2/3) - Enzymes
4

Cell Membranes & Transport

4.1

Biological Membranes

4.1.1Fluid Mosaic Model4.1.2Cell Signalling4.1.3End of Topic Test - Biological Membranes4.1.4Exam-Style Question - Membranes
4.2

Movement Into & Out of Cells

4.2.1Passive Movement Across the Membrane4.2.2Active Movement Across the Membrane4.2.3Osmosis4.2.4Osmosis & Plant Cells4.2.5Surface Area to Volume Ratio4.2.6Adaptions to Surface Area to Volume Ratio4.2.7Calculations - Volume4.2.8Calculations - Surface Area
5

The Mitotic Cell Cycle

5.1

Replication & Division of Nuclei

5.1.1Chromosomes5.1.2The Cell Cycle5.1.3Mitosis5.1.4Role of Mitosis5.1.5Stem Cells5.1.6Mitosis & Cancer5.1.7End of Topic Test - Replication & Division5.1.8A-A* (AO2/3) - Cell Cycle & Transport
6

Nucleic Acids & Protein Synthesis

6.1

Nucleic Acids

6.1.1Introduction to DNA & RNA6.1.2Nucleotides6.1.3Polynucleotides6.1.4ADP & ATP6.1.5DNA6.1.6DNA Replication6.1.7RNA6.1.8End of Topic Test - Nucleic Acids & ATP
6.2

Protein Synthesis

6.2.1The Genetic Code6.2.2Polypeptide Synthesis6.2.3RNA Processing in Eukaryotes6.2.4Mutations6.2.5Exam-Style Question - Protein Synthesis6.2.6A-A* (AO2/3) - Coronavirus Translation
7

Transport in Plants

7.1

Transport in Plants

7.1.1Plant Vessels7.1.2Xylem7.1.3Investigating Transpiration7.1.4Adaptations to Water Availability7.1.5Phloem7.1.6End of Topic Test - Transport in Animals & Plants7.1.7A-A* (AO2/3) - Transport in Animals & Plants
8

Transport in Mammals

8.1

Circulatory System

8.1.1Double Circulation8.1.2Blood Vessels8.1.3Important Blood Vessels8.1.4Blood Cells8.1.5Tissue Fluid
8.2

Transport of Oxygen & Carbon Dioxide

8.2.1Haemoglobin8.2.2Oxygen Dissociation
8.3

The Heart

8.3.1Structure of the Heart8.3.2The Cardiac Cycle8.3.3Coordination of Heart Action
9

Gas Exchange

9.1

Gas Exchange System

9.1.1Mammalian Gas Exchange9.1.2Smoking
10

Infectious Diseases

10.1

Infectious Diseases

10.1.1Transmission of Disease10.1.2Tuberculosis10.1.3HIV10.1.4Malaria10.1.5Cholera10.1.6Measles
10.2

Antibiotics

10.2.1Antibiotics
11

Immunity

11.1

The Immune System

11.1.1Phagocytes11.1.2Antigens11.1.3Lymphocytes11.1.4Primary & Secondary Response
11.2

Antibodies & Vaccination

11.2.1Antibodies11.2.2Monoclonal Antibodies11.2.3Immunity11.2.4Vaccines11.2.5Exam-Style Question - Immune System11.2.6A-A* (AO2/3) - Disease & Immune System
12

Energy & Respiration (A2 Only)

12.1

Energy

12.1.1Energy & ATP12.1.2Respiratory Substrates & Quotient12.1.3Respirometers
12.2

Respiration

12.2.1Introduction to Respiration12.2.2Anaerobic Respiration12.2.3Aerobic Respiration12.2.4Chemiosmosis & Coenzymes12.2.5Mitochondria12.2.6Respiration Experiments12.2.7End of Topic Test - Respiration12.2.8A-A* (AO3/4) - Respiration
13

Photosynthesis (A2 Only)

13.1

Photosynthesis

13.1.1Introduction to Photosynthesis13.1.2Light-Dependent Reactions13.1.3Light-Independent Reactions13.1.4Limiting Factors13.1.5Investigating Photosynthesis13.1.6End of Topic Test - Photosynthesis13.1.7A-A* (AO3/4) - Photosynthesis
14

Homeostasis (A2 Only)

14.1

Homeostasis

14.1.1Homeostasis14.1.2Negative Feedback
14.2

The Kidney

14.2.1Kidneys & Osmoregulation
14.3

Cell Signalling

14.3.1Messengers & cAMP
14.4

Blood Glucose Concentration

14.4.1Blood Glucose Concentration14.4.2Controlling Blood Glucose14.4.3Urinalysis
14.5

Homeostasis in Plants

14.5.1Stomata & Guard Cells
15

Control & Coordination (A2 Only)

15.1

Control & Coordination in Mammals

15.1.1Neurones15.1.2Receptors15.1.3Taste15.1.4Reflexes15.1.5Action Potentials15.1.6Saltatory Conduction15.1.7Synapses15.1.8Cholinergic Synnapses15.1.9Neuromuscular Junction15.1.10Skeletal Muscle15.1.11Sliding Filament Theory Contraction15.1.12Sliding Filament Theory Contraction 215.1.13Menstruation15.1.14Contraceptive Pill
15.2

Control & Co-Ordination in Plants

15.2.1Venus Flytrap15.2.2Auixns15.2.3Gibberellins
16

Inherited Change (A2 Only)

16.1

Passage of Information to Offspring

16.1.1Meiosis16.1.2Gametogenesis16.1.3Meiosis & Genetic Diversity
16.2

Genes & Phenotype

16.2.1Key Terms in Genetics16.2.2Inheritance16.2.3Linkage16.2.4Multiple Alleles16.2.5Chi-Squared Test16.2.6Mutations16.2.7Specific Mutations16.2.8End of Topic Test - Genetics16.2.9A-A* (AO2/3) - Genetics
16.3

Gene Control

16.3.1lac Operon16.3.2Transcription Factors16.3.3Gibberellins & DELLA Proteins
17

Selection & Evolution (A2 Only)

17.1

Variation

17.1.1Phenotypic Variation17.1.2Continuous & Discontinuous Variation17.1.3t-Tests
17.2

Natural & Artificial Selection

17.2.1Natural Selection & Evolution17.2.2Types of Selection17.2.3Types of Selection Summary17.2.4Genetic Drift & Founder Effect17.2.5Populations17.2.6Hardy-Weinberg Principle17.2.7Artificial Selection
17.3

Evolution

17.3.1Evolution by Natural Selection17.3.2Evidence for Evolution17.3.3Speciation17.3.4Extinction
18

Classification & Conservation (A2 Only)

18.1

Biodiversity

18.1.1Key Terms18.1.2Biodiversity18.1.3Sampling Methods18.1.4Correlation18.1.5Spearman’s Rank18.1.6Measuring Biodiversity
18.2

Classification

18.2.13-Domain Classification System18.2.2Linnaean Classification System
18.3

Conservation

18.3.1Importance of Biodiversity18.3.2Conservation18.3.3Zoos, Seedbanks, & Other Conservation Methods18.3.4Assisted Reproduction & Contraception18.3.5Non-Governmental Organisations
19

Genetic Technology (A2 Only)

19.1

Manipulating Genomes

19.1.1Recombinant DNA & Genetic Engineering19.1.2Restriction Endonucleases19.1.3Gel Electrophoresis19.1.4DNA Probes & Microarrays19.1.5End of Topic Test - Manipulating Genomes19.1.6A-A* (AO2/3) - Manipulating Genomes
19.2

Genetic Technology Applied to Medicine

19.2.1Bioinformatics19.2.2Applications of Recombinant DNA19.2.3Genetic Screening19.2.4Gene Therapy19.2.5Ethics19.2.6DNA Profiling & Forensics
19.3

Genetically Modified Organisms in Agriculture

19.3.1GM Crops & Livestock19.3.2Ethics

Practice questions on Gene Therapy

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  1. 1
    Which of the following statements about gene therapy are true?True / false
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Genetic Screening

Ethics