Genetic Therapy and Stem Cells Review

These flashcards cover key concepts related to genetic therapy and stem cells, focusing on definitions, comparisons, applications, and ethical considerations in genetic disease modeling.

What are the two main types of gene therapy

Ex vivo gene therapy and in vivo gene therapy.

What are some vectors used for delivering genetic materials?

Non-viral and viral vectors.

What is the role of pluripotent stem cells in research?

They are used for in vitro models and can differentiate into various cell types.

What are organoid cultures?

Three-dimensional cultures that involve co-cultures of different cell types.

What are the advantages of using cellular models over animal models?

Faster analysis, lower costs, and no ethical concerns.

What are the disadvantages of cellular models?

Limited range of analyses, lack of cellular environment, and limited variety of cells.

What do the '3R' principles refer to?

Refine, Reduce, Replace animal experiments.

What is therapeutic cloning?

Transplantation of a cell nucleus into an enucleated egg for generating pluripotent stem cells.

What factors are used to induce pluripotency in adult cells?

OSKM: OCT3/4, SOX2, KLF4, MYC.

What can induced pluripotent stem cells (iPSCs) be used for?

Autologous cell therapy, ex vivo gene therapy, drug screening, and modeling human diseases.

Can pluripotent stem cells be generated from adult cells without using embryonic material?

Yes, through reprogramming of somatic cells.

What is the significance of co-culture systems?

They allow the interaction of different cell types to study pathophysiological contexts.

What is the importance of genetic therapy for monogenic diseases?

It can involve protein replacement, substrate removal, and small molecules.

What does in vivo gene therapy utilize?

Adenovirus and adeno-associated virus.

How are mutant pluripotent stem cells created?

By culturing cells from mutation carriers and performing gene editing.

What applications can differentiation of pluripotent stem cells have?

They can generate dopaminergic neurons, cardiomyocytes, and pancreatic precursors.

What is a hemapoietic stem cell?

A type of stem cell involved in ex vivo gene therapy.

What are some examples of types of cellular models?

Monolayer cultures, 3D organoid cultures, and transgenic models.

What challenges are commonly faced when developing cellular models?

Generating sufficient numbers of cells and overcoming limitations of single cell type analysis.

What characterizes embryonic stem cells (ES)?

They can differentiate into cells from all three germ layers: ectoderm, mesoderm, endoderm.

What does gene therapy aim to achieve?

Correcting genetic disorders by delivering functional genes.

What ethical concerns are associated with gene therapy?

Potential implications of genome cloning and targeting.

What are organoids used for in disease modeling?

They simulate the architecture and functionality of human organs for research and drug testing.

What is defined differentiation in the context of stem cells?

Guiding pluripotent stem cells to mature into specific cell types using growth factors.

What is a major limitation of using iPSCs in therapy?

The risk of tumorigenesis and cell rejection.

What is the role of transcription factors in generating iPSCs?

They maintain pluripotency and allow differentiation of stem cells.

What are patient-derived models used for?

They help understand genetic diseases at the cellular level.

What is a monoculture system?

A culture system comprising only one type of cell, limiting cellular interaction.

What type of genetic approach might involve germ-line gene therapy?

Altering genes in sperm or eggs to affect future generations.

What factor is involved in the mechanism of action of pluripotency?

OCT3/4, which is essential for maintaining stem cell pluripotency.

Why are animal models sometimes necessary despite the benefits of cellular models?

To study systemic effects and physiological responses to therapies.

What is a potential consequence of using genetic editing tools on pluripotent cells?

Changes to the genomic integrity that may lead to unexpected outcomes.

How can pluripotent stem cells aid in drug development?

They can be used to screen potential drugs' efficacy and toxicity before clinical trials.