Podcast
Questions and Answers
What is a characteristic of injectable stem cells?
What is a characteristic of injectable stem cells?
- They are more invasive than other methods.
- They allow for controlled growth factor release. (correct)
- They can only grow in one shape.
- They require a surgical implant.
Which of the following materials is not classified as a polymer used in tissue engineering?
Which of the following materials is not classified as a polymer used in tissue engineering?
- Matrigel
- Collagen
- Hydroxyapatite (correct)
- Fibrin
What does solid scaffold manufacturing utilize for accuracy?
What does solid scaffold manufacturing utilize for accuracy?
- Drawing templates.
- Manual sketching techniques.
- 3D printing with standard materials.
- Computer-aided design. (correct)
What innovative approach have researchers used to address the regeneration of human tissues and organs?
What innovative approach have researchers used to address the regeneration of human tissues and organs?
Which of the following is a common material used in the creation of decellularized matrices?
Which of the following is a common material used in the creation of decellularized matrices?
What characterizes cancer at a cellular level?
What characterizes cancer at a cellular level?
Which type of cancer originates from epithelial cells?
Which type of cancer originates from epithelial cells?
What role do mutagens play in the development of cancer?
What role do mutagens play in the development of cancer?
What type of mutations primarily lead to cancer development?
What type of mutations primarily lead to cancer development?
Which of the following is NOT classified as a carcinogen?
Which of the following is NOT classified as a carcinogen?
Which type of cancer is specifically associated with mesoderm cells?
Which type of cancer is specifically associated with mesoderm cells?
What is a major difference between cancer and other genetic diseases?
What is a major difference between cancer and other genetic diseases?
Which characteristic differentiates skin cancer from lung cancer?
Which characteristic differentiates skin cancer from lung cancer?
What is a significant disadvantage of using retroviruses in gene therapy?
What is a significant disadvantage of using retroviruses in gene therapy?
Which characteristic makes adenoviruses advantageous for gene therapy?
Which characteristic makes adenoviruses advantageous for gene therapy?
Which of the following is true about liposomes in gene therapy?
Which of the following is true about liposomes in gene therapy?
What is the method used to identify cancer stem cells (CSCs) mentioned in the content?
What is the method used to identify cancer stem cells (CSCs) mentioned in the content?
What is a disadvantage of using adenoviruses for gene therapy?
What is a disadvantage of using adenoviruses for gene therapy?
Which vector is exclusively used in ex vivo therapy?
Which vector is exclusively used in ex vivo therapy?
Which surface marker is specifically known as prominin-1?
Which surface marker is specifically known as prominin-1?
What is a common disadvantage associated with liposomes in gene therapy?
What is a common disadvantage associated with liposomes in gene therapy?
What is one of the roles of CD44 as described in the content?
What is one of the roles of CD44 as described in the content?
How do retroviruses achieve stable modification of target cells?
How do retroviruses achieve stable modification of target cells?
Which intracellular marker is NOT mentioned as a marker for cancer stem cells?
Which intracellular marker is NOT mentioned as a marker for cancer stem cells?
What is a significant limitation of using cellular markers for isolating cancer stem cells?
What is a significant limitation of using cellular markers for isolating cancer stem cells?
Which gene therapy vector does not facilitate uncontrolled integration and has non-integration to the chromosome as an advantage?
Which gene therapy vector does not facilitate uncontrolled integration and has non-integration to the chromosome as an advantage?
Which of the following is a role of EpCAM as stated in the content?
Which of the following is a role of EpCAM as stated in the content?
Which of the following is NOT a characteristic of immature cells in culture?
Which of the following is NOT a characteristic of immature cells in culture?
Which protein is associated with oncogenic potential as mentioned in the content?
Which protein is associated with oncogenic potential as mentioned in the content?
What effect did the therapy have on the immune system?
What effect did the therapy have on the immune system?
What is a significant consequence of ammonia accumulation in the blood for individuals with Ornithine transcarbamylase deficiency?
What is a significant consequence of ammonia accumulation in the blood for individuals with Ornithine transcarbamylase deficiency?
Why were all gene therapy trials using retroviral vectors in blood stem cells halted by the FDA in 2003?
Why were all gene therapy trials using retroviral vectors in blood stem cells halted by the FDA in 2003?
For what condition was the stem cell gene therapy Strimvelis designed?
For what condition was the stem cell gene therapy Strimvelis designed?
Which therapeutic gene-targeting strategy can be utilized against HIV?
Which therapeutic gene-targeting strategy can be utilized against HIV?
What aspect of gene therapy in blood cells was demonstrated by the prior study treating bubble baby syndrome?
What aspect of gene therapy in blood cells was demonstrated by the prior study treating bubble baby syndrome?
Which of the following best describes the nature of the enzyme production in the mentioned gene therapy?
Which of the following best describes the nature of the enzyme production in the mentioned gene therapy?
Which of the following diseases can be potentially treated using similar therapeutic strategies as mentioned?
Which of the following diseases can be potentially treated using similar therapeutic strategies as mentioned?
What is the primary objective of tissue engineering?
What is the primary objective of tissue engineering?
Which type of artificial tissue has been approved by the FDA for limited use in human patients?
Which type of artificial tissue has been approved by the FDA for limited use in human patients?
What challenge has hindered the expansion of cells in tissue engineering?
What challenge has hindered the expansion of cells in tissue engineering?
Which of the following is a component of tissue engineering?
Which of the following is a component of tissue engineering?
What materials have been used to create artificial skin in successful medical applications?
What materials have been used to create artificial skin in successful medical applications?
What is a major factor that has limited clinical advancements in regenerative medicine?
What is a major factor that has limited clinical advancements in regenerative medicine?
For how long has tissue engineering been a field of research?
For how long has tissue engineering been a field of research?
Why was it difficult to expand most human cells in vitro during the early 1990s?
Why was it difficult to expand most human cells in vitro during the early 1990s?
Flashcards
Injectable stem cells
Injectable stem cells
A method of delivering stem cells into the body by injecting them directly into the desired area. This technique is less invasive than other methods and allows the cells to adapt to their surrounding environment.
Decellularized matrix
Decellularized matrix
A scaffold created from the naturally occurring extracellular matrix (ECM) of an organ. This material is biocompatible and provides a framework for new tissue growth.
Biomaterial
Biomaterial
A biological material that serves as a template or framework for tissue regeneration. These are derived from natural sources like collagen or ECM.
Solid scaffold manufacturing
Solid scaffold manufacturing
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Decellularization
Decellularization
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Tissue Engineering
Tissue Engineering
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Goal of Tissue Engineering
Goal of Tissue Engineering
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Regenerative Medicine
Regenerative Medicine
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Inability to Expand Cells In Vitro
Inability to Expand Cells In Vitro
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In Vitro
In Vitro
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Inadequate Vascularity
Inadequate Vascularity
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Scaffolds
Scaffolds
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Biologically Active Molecules
Biologically Active Molecules
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What is cancer?
What is cancer?
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What are carcinomas?
What are carcinomas?
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What are sarcomas?
What are sarcomas?
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What are adenocarcinomas?
What are adenocarcinomas?
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What are carcinogens?
What are carcinogens?
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What are mutagens?
What are mutagens?
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Cancer is a disease of the genome at the cellular level.
Cancer is a disease of the genome at the cellular level.
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What are the genetic alterations in cancer?
What are the genetic alterations in cancer?
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Retroviruses
Retroviruses
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Adenoviruses
Adenoviruses
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Liposomes
Liposomes
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Viral vectors and cancer risk
Viral vectors and cancer risk
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Retroviruses and stable modification
Retroviruses and stable modification
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Adenovirus and transient gene expression
Adenovirus and transient gene expression
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Liposome inefficiency
Liposome inefficiency
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Liposome and transient gene expression
Liposome and transient gene expression
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In vivo gene therapy
In vivo gene therapy
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Ornithine transcarbamylase deficiency (OTCD)
Ornithine transcarbamylase deficiency (OTCD)
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Gene therapy in blood cells
Gene therapy in blood cells
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X-linked severe combined immunodeficiency disease (SCID)
X-linked severe combined immunodeficiency disease (SCID)
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Therapeutic gene for various diseases
Therapeutic gene for various diseases
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Targets for HIV gene therapy
Targets for HIV gene therapy
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Strimvelis
Strimvelis
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Gene therapy
Gene therapy
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Spheroid Colony Formation
Spheroid Colony Formation
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CD133
CD133
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CD44
CD44
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EpCAM
EpCAM
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Aldehyde Dehydrogenase 1 (ALDH1)
Aldehyde Dehydrogenase 1 (ALDH1)
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Oct3/4
Oct3/4
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Nanog
Nanog
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Isolation of CSCs with Markers
Isolation of CSCs with Markers
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Study Notes
Stem Cells in Gene Therapy/Gene Editing
- Cell/gene therapy is a branch of Regenerative Medicine focused on replacing, engineering, or regenerating human cells, tissues, or organs to restore normal function.
- Gene therapy delivers therapeutic genes into a patient's cells to treat disease.
- Cell therapy delivers intact, living cells into a patient to treat disease.
- Combination cell/gene therapy approaches aim to insert genes into a patient's own cells to control diseases, and are now in clinical trials.
Gene Therapy
- Gene therapy is an experimental technique using genes to treat or prevent diseases.
- Future approaches may involve inserting genes into patient cells instead of drugs or surgery.
- Several gene therapy approaches are being researched, including:
- Replacing mutated disease-causing genes with healthy copies.
- Inactivating, or "knocking out," mutated genes that function improperly.
- Introducing a new gene to fight a disease.
- Although promising, gene therapy is still risky and under study for safety and effectiveness.
- Current gene therapy trials are focused on diseases with no other known cures.
Different Routes of Gene Therapy
- Ex vivo approach:
- Target cells are removed from the body and grown in a lab (in vitro).
- Genes are introduced into the cultured cells.
- The modified cells are returned to the patient.
- Examples: fibroblasts, hematopoietic cells, stem cells
- In vivo approach (Direct Gene Transfer):
- Genes are introduced directly into affected tissue of the body without removing cells.
- Special delivery vehicles are typically required for gene transfer
- Examples: lungs, brain
Ex Vivo and In Vivo Gene Therapies
- Ex vivo gene therapy is often used with blood cells (lymphocytes or hematopoietic stem cells) for conditions affecting the hematopoietic system.
- In vivo gene therapy is used in cancer treatment using oncolytic adenoviruses, and in muscular dystrophy or hemophilia treatment with adeno-associated vectors. In other instances, non-viral vectors are used since they are not oncogenic.
Ex Vivo Gene Therapy: Putting Functional Genes Into Marrow Stem Cells
- This approach involves removing stem cells from the patients bone marrow or blood, modifying them in a lab, and then reintroducing them to the patient.
Methods of Gene Delivery
- Physical methods:
- Parenteral injection
- Microinjection
- Aerosol
- Gene gun
- Chemical methods:
- Calcium phosphate
- DEAE-Dextran
- Liposomes
- Biological methods:
- Viral vectors (retrovirus, adenovirus, HSV)
Common Vectors Used For Gene Therapy
- Retroviruses:
- Used in ex vivo therapy only.
- Advantages: chromosomal integration and stable modification of target cells.
- Disadvantages: uncontrolled integration, potential oncogenicity, and inability to infect non-dividing cells.
- Adenoviruses:
- Second most common delivery system, which can be produced in large quantities in cultures.
- Advantages: infect non-dividing cells, non-integration.
- Disadvantages: transient expression due to episomal integration and may provoke an immune response.
- Liposomes:
- Lipid bilayers surrounding an aqueous vesicle that can carry large DNA.
- Advantages: safer than viral vectors, can carry large DNA molecules.
- Disadvantages: inefficient and transient gene transfer.
Somatic Cell Therapy
- Gene insertion into somatic cells (fibroblasts, myoblasts, epithelial cells, etc.)
- Corrects the genetic defect in the patient; However the change is not inheritable.
Germ Line Therapy
- Introducing the foreign gene into germ cells (sperm, ovum, fertilized egg)
- Modifies features in both somatic and germ cells (heritable changes).
- Considered unethical and not advocated in current use.
History of Gene Therapy
- Early clinical trials started in the 1980s and a patient died in a 1990s trial.
- The first report of a human gene therapy trial involving stem cells started in the 1990s involving treatment of a rare inherited genetic disease.
- In 2003, FDA halted trials using retroviral vectors in blood stem cells.
Some Targets for Gene Therapy
- Gene therapy can be used to treat/prevent diseases such as cancer or genetic disorders. Infections are also being targeted.
Gene Therapy in Blood Cells (Hematopoietic Stem Cells)
- Blood stem cells are a target for gene therapies that can treat various conditions of the hematopoietic system, and often using ex vivo approaches.
Somatic Cell Therapy History
- The first human gene transfer using stem cells was performed in 1990.
Stem Cells and Tissue Engineering
- Tissue engineering is a field that combines scaffolds, cells, and biologically active molecules to create functional tissues.
- The goal is to restore, maintain, or improve damaged tissues and organs.
- Examples include artificial skin and cartilage, but clinical applications are frequently limited.
Regenerative Medicine
- Regenerative medicine focuses on enabling the body to heal itself through tissue engineering, and cell therapy and regeneration.
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