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Deciphering the Role of Gene Therapy in Regenerative Medicine

Description: Deciphering the Role of Gene Therapy in Regenerative Medicine
Number of Questions: 15
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Tags: gene therapy regenerative medicine genetic engineering cell therapy
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What is the primary goal of gene therapy in regenerative medicine?

  1. To introduce genetic material into cells to correct genetic defects or enhance cellular functions.

  2. To replace damaged or diseased tissues with healthy ones.

  3. To stimulate the body's natural healing mechanisms.

  4. To prevent the development of genetic disorders.


Correct Option: A
Explanation:

Gene therapy aims to introduce genetic material into cells to correct genetic defects or enhance cellular functions, thereby promoting tissue regeneration and repair.

Which type of gene therapy involves the transfer of a functional gene into cells to replace a defective one?

  1. Gene augmentation

  2. Gene editing

  3. Gene silencing

  4. Gene replacement


Correct Option: D
Explanation:

Gene replacement therapy involves transferring a functional copy of a gene into cells to replace a defective or missing gene.

What is the main advantage of using viral vectors in gene therapy?

  1. They can efficiently deliver genetic material into cells.

  2. They are less likely to cause an immune response.

  3. They can integrate the genetic material into the host cell's genome.

  4. They are easy to produce and purify.


Correct Option: A
Explanation:

Viral vectors are commonly used in gene therapy because they can efficiently deliver genetic material into cells, enabling the introduction of therapeutic genes.

Which gene therapy approach aims to inactivate or suppress the expression of a specific gene?

  1. Gene editing

  2. Gene silencing

  3. Gene augmentation

  4. Gene replacement


Correct Option: B
Explanation:

Gene silencing approaches, such as RNA interference (RNAi) and CRISPR interference (CRISPRi), aim to inactivate or suppress the expression of a specific gene by targeting its mRNA or DNA.

What is the primary challenge associated with the use of viral vectors in gene therapy?

  1. The risk of insertional mutagenesis

  2. The potential for immune responses

  3. The difficulty in targeting specific cells

  4. The high cost of production


Correct Option: A
Explanation:

A major challenge associated with the use of viral vectors in gene therapy is the risk of insertional mutagenesis, where the integration of the genetic material into the host cell's genome can disrupt gene function or lead to tumor formation.

Which gene therapy technique involves the precise modification of a gene's sequence to correct a genetic defect?

  1. Gene editing

  2. Gene silencing

  3. Gene augmentation

  4. Gene replacement


Correct Option: A
Explanation:

Gene editing techniques, such as CRISPR-Cas9 and TALENs, allow for precise modification of a gene's sequence, enabling the correction of genetic defects or the introduction of desired genetic changes.

What is the main goal of gene therapy in the context of regenerative medicine?

  1. To stimulate tissue regeneration and repair

  2. To prevent the development of genetic disorders

  3. To replace damaged or diseased tissues with healthy ones

  4. To enhance immune function


Correct Option: A
Explanation:

In regenerative medicine, gene therapy aims to stimulate tissue regeneration and repair by introducing therapeutic genes into cells or tissues, promoting the growth and function of new tissue.

Which type of gene therapy involves the transfer of a gene into cells to enhance their function or confer new properties?

  1. Gene augmentation

  2. Gene editing

  3. Gene silencing

  4. Gene replacement


Correct Option: A
Explanation:

Gene augmentation therapy involves transferring a gene into cells to enhance their function or confer new properties, such as resistance to disease or improved tissue regeneration.

What is a potential concern associated with the use of gene therapy in regenerative medicine?

  1. The risk of off-target effects

  2. The potential for immune responses

  3. The difficulty in delivering genetic material to specific cells

  4. All of the above


Correct Option: D
Explanation:

Gene therapy in regenerative medicine faces several potential concerns, including the risk of off-target effects, the potential for immune responses, and the difficulty in delivering genetic material to specific cells.

Which gene therapy approach involves the use of small interfering RNAs (siRNAs) to silence the expression of a specific gene?

  1. Gene editing

  2. Gene silencing

  3. Gene augmentation

  4. Gene replacement


Correct Option: B
Explanation:

Gene silencing approaches, such as RNA interference (RNAi) using siRNAs, involve the use of small interfering RNAs to silence the expression of a specific gene by targeting its mRNA.

What is the main challenge associated with the use of non-viral vectors in gene therapy?

  1. The risk of insertional mutagenesis

  2. The potential for immune responses

  3. The difficulty in targeting specific cells

  4. The low efficiency of gene delivery


Correct Option: D
Explanation:

Non-viral vectors, such as liposomes and nanoparticles, often face challenges in achieving efficient delivery of genetic material into cells, limiting their effectiveness in gene therapy.

Which gene therapy approach involves the use of zinc finger nucleases (ZFNs) to modify a gene's sequence?

  1. Gene editing

  2. Gene silencing

  3. Gene augmentation

  4. Gene replacement


Correct Option: A
Explanation:

Gene editing approaches, such as zinc finger nucleases (ZFNs), allow for precise modification of a gene's sequence, enabling the correction of genetic defects or the introduction of desired genetic changes.

What is the primary goal of gene therapy in the context of regenerative medicine?

  1. To stimulate tissue regeneration and repair

  2. To prevent the development of genetic disorders

  3. To replace damaged or diseased tissues with healthy ones

  4. To enhance immune function


Correct Option: A
Explanation:

In regenerative medicine, gene therapy aims to stimulate tissue regeneration and repair by introducing therapeutic genes into cells or tissues, promoting the growth and function of new tissue.

Which type of gene therapy involves the transfer of a gene into cells to enhance their function or confer new properties?

  1. Gene augmentation

  2. Gene editing

  3. Gene silencing

  4. Gene replacement


Correct Option: A
Explanation:

Gene augmentation therapy involves transferring a gene into cells to enhance their function or confer new properties, such as resistance to disease or improved tissue regeneration.

What is a potential concern associated with the use of gene therapy in regenerative medicine?

  1. The risk of off-target effects

  2. The potential for immune responses

  3. The difficulty in delivering genetic material to specific cells

  4. All of the above


Correct Option: D
Explanation:

Gene therapy in regenerative medicine faces several potential concerns, including the risk of off-target effects, the potential for immune responses, and the difficulty in delivering genetic material to specific cells.

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