Gene Therapy Vector Delivery Systems

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Questions and Answers

What is the primary goal of gene therapy?

To eliminate all genetic disorders

To cure only blood disorders

To prevent future generations from inheriting genetic diseases

To replace, manipulate, or supplement nonfunctional or malfunctioning genes with healthy genes (correct)

What is a major drawback of gene therapy?

It is a very new field of research

It is a complex procedure

It is extremely expensive (correct)

It has no cure rate

What type of gene therapy aims to prevent genetic diseases from affecting future generations?

Gene manipulation

Germline gene therapy (correct)

Gene editing

Somatic gene therapy

What is a benefit of gene therapy?

It provides treatments for previously incurable diseases Signup and view all the answers

What is the function of genes?

To encode instructions for making proteins Signup and view all the answers

What type of gene therapy focuses on treating an individual's existing condition?

Somatic gene therapy Signup and view all the answers

What is a potential application of gene therapy?

All of the above Signup and view all the answers

What is the expected outcome of gene therapy in the future?

It will become more affordable Signup and view all the answers

What is the main objective of Somatic Gene Therapy?

To introduce a functional ADA gene into the patient's immune cells Signup and view all the answers

What is the goal of Germline Gene Therapy?

To edit faulty genes in embryos to prevent genetic disorders Signup and view all the answers

What is the primary concern with Germline Gene Therapy?

High ethical concerns Signup and view all the answers

What is the characteristic of Somatic Gene Therapy?

Effects are short-lived and limited to the individual Signup and view all the answers

What is the advantage of Somatic Gene Therapy?

It has less ethical concerns Signup and view all the answers

What is an example of a genetic disorder that can be prevented using Germline Gene Therapy?

Cystic fibrosis Signup and view all the answers

What is the main goal of Gene Augmentation therapy?

To introduce a new protein coding gene into somatic cells Signup and view all the answers

What is a characteristic of Gene Augmentation therapy?

It is a straightforward strategy Signup and view all the answers

What is a characteristic of Adeno-Associated Viruses (AAVs)?

They can integrate their genetic material into a specific location on a chromosome. Signup and view all the answers

What is the main limitation of using electroporation for gene transfer?

It can cause high rates of cell death. Signup and view all the answers

What is the purpose of using a mutant HSV-1 strain for gene transfer?

To reduce the risk of lytic cycle of viral replication. Signup and view all the answers

What is the mechanism of gene gun for DNA transfer?

It uses gold particles coated with DNA to enter cells. Signup and view all the answers

Why are adenoviruses used in gene therapy?

They are used as auxiliary viruses for AAVs. Signup and view all the answers

What is the advantage of using Herpes Simplex Viruses (HSV) for gene transfer?

They have a reduced risk of insertional mutagenesis. Signup and view all the answers

What is the function of electroporation in gene transfer?

To form temporary pores in the cell membrane. Signup and view all the answers

What is a characteristic of tissue tropism?

It is the ability of a virus to infect a specific cell type. Signup and view all the answers

What is the primary disadvantage of using retroviruses as gene therapy vectors?

They can cause insertional mutagenesis Signup and view all the answers

What is the key difference between in-vivo and ex-vivo gene therapy?

In-vivo involves direct vector introduction, while ex-vivo involves indirect vector introduction Signup and view all the answers

What is the characteristic of adenoviruses that makes them useful for gene therapy?

They can introduce their genetic material into the host cell nucleus without integrating into the host genome Signup and view all the answers

What is the advantage of using retroviruses as gene therapy vectors?

They are able to integrate their genome into the host cell genome Signup and view all the answers

What is the primary concern with using in-vivo gene therapy?

Off-target effects Signup and view all the answers

What is the characteristic of retroviruses that makes them a subclass of RNA viruses?

They have a reverse transcriptase enzyme Signup and view all the answers

What is the primary advantage of using ex-vivo gene therapy?

It allows for more control over gene expression Signup and view all the answers

What is the characteristic of adenoviruses that distinguishes them from retroviruses?

They have a double-stranded DNA genome Signup and view all the answers

What is the primary cause of Parkinson's disease?

Absence of a chemical called GABA in the brain Signup and view all the answers

What is the function of the gene that produces glutamic acid decarboxylase (GAD) in gene therapy for Parkinson's disease?

To increase the level of GABA in the brain Signup and view all the answers

What is the primary cause of Alzheimer's disease?

Reduced levels of BDNF in damaged brain regions Signup and view all the answers

What is the goal of gene therapy in Alzheimer's disease?

To boost the levels of BDNF in damaged brain regions Signup and view all the answers

What is the advantage of direct delivery of the cystic fibrosis transmembrane regulator (CFTR) gene into respiratory tract epithelium cells?

It can bypass the physical and immunity barriers in the host's lung Signup and view all the answers

What is the challenge of gene therapy for cystic fibrosis?

The physical and immunity barriers in the host's lung Signup and view all the answers

What type of vector system is the most advanced for cystic fibrosis gene therapy?

Adenovectors Signup and view all the answers

What is the goal of gene therapy for cystic fibrosis?

To deliver the cystic fibrosis transmembrane regulator (CFTR) gene to respiratory tract epithelium cells Signup and view all the answers

Study Notes

Routes of Administration

In-vivo (Direct): introduces vectors directly into the body, less invasive, simpler, and has more off-target effects
Ex-vivo (Cell-based): introduces vectors indirectly, more invasive, more complex, and has more control

Gene Therapy

Definition: an experimental procedure aimed at replacing, manipulating, or supplementing nonfunctional or malfunctioning genes with healthy genes
Goal: to treat genetic disorders that previously had no conventional medical treatment
Recent developments: effective and long-term cured cases have been reported, but high cost is a major drawback

Types of Gene Therapy

Somatic Gene Therapy: focuses on treating an individual's faulty condition, effects are short-lived, and has less ethical concerns
Germline Gene Therapy: aims to prevent genetic diseases from affecting future generations, makes permanent changes, and has high ethical concerns

Gene Therapy Applications

Severe Combined Immunodeficiency (SCID): somatic gene therapy introduces a functional ADA gene into immune cells
Preventing Inherited Genetic Disorders: germline gene therapy edits the faulty gene in embryos to prevent the disorder from being passed on to children

Gene Therapy Strategies

Gene Augmentation Therapy: introduces a new protein-coding or functional gene to enhance cell functionality
Gene Inhibition Therapy: reduces or eliminates the expression of a gene
Gene Editing Therapy: makes precise changes to a gene
Suicide Gene Therapy: induces cell death in target cells

Viral Vectors

Adenovirus: has a double-stranded DNA genome, causes respiratory, intestinal, and ocular infections, and can be used for gene therapy
Adeno-Associated Virus (AAV): a small, single-stranded DNA virus, non-pathogenic, and can insert genetic material into a specific location on a chromosome
Herpes Simplex Virus (HSV): a dsDNA human neurotropic virus used for gene transfer, mainly in the nervous system
Retrovirus: can convert RNA genomes into double-stranded DNA and integrate into host cell chromosomes, but has the risk of insertional mutagenesis

Physical Methods of Gene Delivery

Electroporation: uses high-voltage short pulses to transfer DNA into cells, but has high rates of cell death
Gene Gun: uses particle bombardment to transfer DNA into cells

Gene Therapy Applications in Diseases

Parkinson's Disease: gene therapy increases the level of GABA in the brain
Alzheimer's Disease: gene therapy boosts levels of BDNF in damaged brain regions
Cystic Fibrosis: direct delivery of the CFTR gene into respiratory tract epithelium cells as the target tissue

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Description

This quiz covers the different routes of administration and delivery systems of gene therapy vectors, including in-vivo and ex-vivo methods, and their characteristics.