Human Gene therapy Flashcards
(21 cards)
What is human gene therapy?
A medical approach that aims to treat or prevent diseases by correcting underlying genetic problems.
How does gene therapy work?
By altering a person’s genetic makeup through gene transfer/addition or genome editing.
What is gene transfer/addition?
Introducing a new, healthy gene into cells to replace a faulty one or help fight a disease.
What is genome editing?
Precisely correcting or making targeted changes to DNA in cells to restore function.
What is somatic cell gene therapy?
Modifies genes in non-reproductive cells, meaning changes are not passed to future generations.
What is germline gene therapy?
Modifies genes in reproductive cells, meaning changes can be passed to future generations.
What types of diseases can gene therapy treat?
Genetic diseases, cancer, infectious diseases.
What are viral vectors?
Modified viruses used to deliver therapeutic genes into cells.
What is plasmid DNA?
Circular DNA molecules that can be genetically engineered to carry therapeutic genes into human cells.
What is a potential benefit of gene therapy?
Offers the possibility of a permanent cure for genetic diseases.
What are some diseases gene therapy has shown promise in treating?
Neuromuscular disorders, cancer, blindness.
What is one potential application of gene therapy for infants?
Restoring vision for infants with serious vision loss from retinal diseases.
What is non-integrating gene therapy?
An approach that does not disrupt the rest of the genome, reducing side effect risks.
What is a risk associated with gene therapy?
Genetic damage and immune system activation can occur.
What are the ethical concerns surrounding gene therapy?
Potential for misuse in ‘designer babies’ and high therapy costs.
What is a challenge regarding the cost of gene therapy?
The high cost can limit access for many patients.
Is gene therapy a guaranteed cure?
No, it is not guaranteed and may have unexpected side effects.
What complicates the delivery of gene therapy?
The potential for an immune response to foreign material.
What is a potential security risk associated with gene therapy?
The ability to engineer pathogens or create genetically modified organisms for malicious purposes.
What is a concern regarding human diversity and gene therapy?
It could lead to a reduction in human diversity and lack of natural selection.
What is the risk of losing a sense of self in gene therapy?
Particularly in the context of gene therapy for the brain or changes in sexual orientation.
