Gene Therapy Basics Quiz

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What is the main goal of gene therapy?

To treat loss-of-function genetic disorders by delivering therapeutic DNA sequences into the nucleus of a cell (correct)

To introduce genes into somatic cells for ethical considerations

To alter the genetic makeup of germ cells

To amplify and isolate normal genes

What is the difference between germ-line gene therapy and somatic gene therapy?

Germ-line gene therapy introduces genes into somatic cells, while somatic gene therapy introduces therapeutic genes into germ-cells

Germ-line gene therapy is used for ethical considerations, while somatic gene therapy is used to alter genetic makeup

Germ-line gene therapy corrects severe disorders, while somatic gene therapy aims to treat loss-of-function genetic disorders

Germ-line gene therapy introduces therapeutic genes into germ-cells or specific embryonal cells, while somatic gene therapy introduces genes into somatic cells (correct)

What happens after germ-line gene therapy is performed?

The therapeutic gene will only be carried by the offspring

The therapeutic gene will not be passed on to the offspring

The therapeutic gene will only be carried by the individual

All cells of the individual derived from these cells will carry the therapeutic gene, including his or her germ cells (correct)

What are the principles of gene therapy?

Isolation and amplification of normal gene, inserting gene into diseased cell, integration into the chromosome or remaining as an extra-chromosomal genetic element Signup and view all the answers

What is the main problem with gene therapy in terms of the longevity of treatment?

Rapid integration of therapeutic DNA into the genome Signup and view all the answers

What was the first disease targeted for gene therapy?

Severe combined immunodeficiency (SCID) Signup and view all the answers

What is Gendicine designed to treat?

Tumors with mutated p53 genes Signup and view all the answers

What is the main limitation of direct injection as a gene therapy delivery method?

Low level of stable integration of DNA Signup and view all the answers

Which gene therapy approach involves the use of the Herpes simplex virus Thymidine kinase (HSV-tk) gene?

Cancer gene therapy Signup and view all the answers

What is the main gene therapy approach for treating Cystic Fibrosis?

In vivo gene therapy Signup and view all the answers

What is the gene therapy approach for treating Duchenne Muscular Dystrophy (DMD)?

Naked plasmid DNA Signup and view all the answers

What is the main challenge in gene therapy for multigene disorders?

Difficulty in introducing more than one gene Signup and view all the answers

Which gene therapy delivery method involves the use of repeated injections and a large number of DNA?

Particle bombardment Signup and view all the answers

What is the main challenge associated with in vivo gene therapy for Cystic Fibrosis?

Inadequate protein production Signup and view all the answers

What is the main challenge associated with naked plasmid DNA gene therapy for Duchenne Muscular Dystrophy (DMD)?

Low level of stable integration of DNA Signup and view all the answers

What is the main challenge associated with suicide gene therapy?

Low level of stable integration of DNA Signup and view all the answers

Gene Therapy Basics Quiz

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Questions and Answers

What is the main goal of gene therapy?

What is the difference between germ-line gene therapy and somatic gene therapy?

What happens after germ-line gene therapy is performed?

What are the principles of gene therapy?

What is the main problem with gene therapy in terms of the longevity of treatment?

What was the first disease targeted for gene therapy?

What is Gendicine designed to treat?

What is the main limitation of direct injection as a gene therapy delivery method?

Which gene therapy approach involves the use of the Herpes simplex virus Thymidine kinase (HSV-tk) gene?

What is the main gene therapy approach for treating Cystic Fibrosis?

What is the gene therapy approach for treating Duchenne Muscular Dystrophy (DMD)?

What is the main challenge in gene therapy for multigene disorders?

Which gene therapy delivery method involves the use of repeated injections and a large number of DNA?

What is the main challenge associated with in vivo gene therapy for Cystic Fibrosis?

What is the main challenge associated with naked plasmid DNA gene therapy for Duchenne Muscular Dystrophy (DMD)?

What is the main challenge associated with suicide gene therapy?

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