Gene Therapy Mechanisms
What molecule stores genetic information in cells?
DNA
Is gene therapy always completely safe?
No
What does “one-and-done” permanent cure mean?
It refers to a single treatment that can permanently fix a genetic problem, eliminating the need for ongoing medication.
Cystic fibrosis is caused by a mutation in the _ gene
CFTR
What is one method used to deliver healthy genes into a patient's cell?
Viruses (viral vectors), Liposomes, Injection
What type of gene therapy raises ethical concerns because changes are passed to future generations?
Germline editing
Why is next-generation gene editing an improvement?
It’s more precise and safer because it can change specific DNA sequences without cutting the entire strand, reducing errors.
What shape do red blood cells take in sickle cell disease?
Sickle, or Crescent shape
What tool is commonly used in gene editing to cut DNA at specific locations?
CRISPR
Why is germline gene editing considered risky?
Because changes are permanent and may have unpredictable effects on future generations
What is in-body gene therapy?
It’s when genes are edited directly inside the patient’s body instead of outside in a lab, making treatment less invasive.
How does gene therapy improve red blood cell function in sickle cell disease?
Restoring the production of normal hemoglobin
What type of gene therapy involves changes that CAN be passed to future generations
Germline Gene Therapy
What serious disease risk can occur if a gene is inserted incorrectly?
Cancer
What are the risks of gene therapy?
Risks include unintended gene changes, immune reactions, and unknown long-term effects.
List at least 2 symptoms caused by cystic fibrosis
inflammation, abdominal pain, nausea, vomiting, mucus in the lungs
When a gene is mutated, what happens?
Mutations can change codons in DNA, causing different amino acids to be added and producing a protein with an altered or nonfunctional structure.
What is the term used to describe genetically enhancing traits like intelligence or appearance?
Designer babies
How could one-and-done cures change healthcare?
They could shift healthcare from managing diseases over time to curing them permanently, reducing the need for ongoing treatments.
What is the main difference between gene therapy for sickle cell disease and cystic fibrosis in modern medicine?
Sickle cell gene therapy is already approved and in use, while cystic fibrosis gene therapy is still mostly experimental and in clinical trials.