Gene Therapy, Genetic Engineering, Stem Cells, and Cloning

These flashcards cover key concepts and details from the lecture on gene therapy, genetic engineering, stem cell research, and cloning.

What is gene therapy?

A technique that modifies genes to treat or prevent diseases.

What are the two primary strategies for gene therapy?

Ex vivo gene therapy and in vivo gene therapy.

What does ex vivo gene therapy involve?

Removing cells from the patient, treating them, and then reintroducing them.

How does in vivo gene therapy differ from ex vivo gene therapy?

It involves directly introducing genes into tissues and organs in the body.

What is transfection?

The introduction of DNA into animal or plant cells.

What are vectors in gene therapy?

Carriers, such as viruses, that deliver therapeutic genes to target cells.

Which type of virus is commonly used as a vector?

Adenovirus, influenza virus, and herpes virus.

What is naked DNA in the context of gene therapy?

DNA injected directly into body tissues without a carrier.

What are liposomes?

Small, hollow particles made of lipid molecules used for gene delivery.

What is RNA interference (RNAi)?

A biological process in which double-stranded RNA inhibits gene expression.

What is the role of small interfering RNAs (siRNAs) in gene silencing?

They bind to target mRNA and lead to its degradation.

What is the CRISPR-Cas9 technology used for?

Genome editing to modify specific genes.

Who won the Nobel Prize in Chemistry in 2020 for CRISPR discoveries?

Emmanuelle Charpentier and Jennifer Doudna.

What disease was successfully treated using CRISPR therapy in 2019?

Sickle Cell Disease (SCD).

What is the function of human embryonic stem cells (hESCs)?

They have the ability to differentiate into any cell type in the body.

Where are human embryonic stem cells derived from?

From the inner cell mass of blastocysts.

What is the difference between adult stem cells and hESCs?

Adult stem cells are tissue-specific and have limited differentiation potential compared to hESCs.

What are induced pluripotent stem cells (iPSCs)?

Adult stem cells reprogrammed to behave like embryonic stem cells.

What is therapeutic cloning?

The process of creating cloned cells for medical purposes without creating a child.

What is the main concern regarding embryonic stem cell research?

Ethical issues surrounding the use of human embryos.

What is the challenge with using adult stem cells?

They are limited in number and differentiation potential.

What is the potential application of stem cells in medicine?

Regenerating damaged tissues and organs.

What are the potential risks of gene therapy?

Adverse reactions, such as infections or immune responses.

What was the outcome of the first human gene therapy in 1990?

It involved treating a SCID patient with adenosine deaminase (ADA) gene therapy.

What are the risks of using viral vectors in gene therapy?

Potential pathogenic effects and immune responses.

What are clonal zygotes used for in cloning?

To replicate genetic material and create identical organisms.

What is reproductive cloning?

Creating a cloned embryo intended to develop into a baby.

What was the first cloned sheep's name?

Dolly.

What is the significance of Dolly the sheep?

She was the first mammal cloned from an adult somatic cell.

What are therapeutic applications of stem cells being researched?

Treatment of leukemia, sickle cell anemia, and repair of damaged organs.

What are the concerns around unregulated stem cell treatments?

Safety, efficacy, and the risk of adverse reactions.

What does CRISPR stand for?

Clustered Regularly Interspaced Short Palindromic Repeats.

What is a major drawback of cloning technologies?

High inefficiency and low success rates.

What ethical concerns are associated with cloning?

Questions regarding the morality and safety of creating genetically identical organisms.

What are organoids?

Miniature organs produced from stem cells for research and therapeutic purposes.

What limitations exist in using gene therapy for diseases?

It is still under study for safety and effectiveness.

What are the uses of CRISPR-Cas9 technology demonstrated in mice?

Removing defective genes responsible for disease.

What is the therapeutic goal of regenerative medicine?

To repair or replace damaged tissues and organs.

Which countries have regulations varying on stem cell research?

Different countries have varying laws; some allow it while others ban it.

What are the genetic benefits of using stem cells for therapies?

They can provide patient-specific treatments minimizing rejection risks.

What is the role of bioengineering in regenerative medicine?

To develop functional tissues and organ structures from stem cells.

What is one promise of stem cell research?

Creating therapies for conditions currently lacking effective treatments.

What is the key to successful gene therapy?

Safe and effective delivery of genetic material to targeted cells.

What are the primary areas of concern for stem cell treatments?

Regulatory issues and potential for unforeseen complications.

What word describes the process of making a copy of a specific gene?

Cloning.

What type of cloning might create tissues matching a patient’s genetics?

Therapeutic cloning.

What is the benefit of three-dimensional bioprinting of tissues?

It allows for the precise construction of functional tissue structures.

What action is taken to prevent immune reactions in organ transplantation?

Creating patient-specific stem cell lines.