Cystic Fibrosis | Pros and Cons! | Solutions for CF |
---|---|---|
What is CFTR
People that suffer from cystic fibrosis have a genetic mutation which inhibits their body to properly create, if even at all, this protein
|
What is Autosomal recessive
The type of inheritance which cystic fibrosis is classified as
|
What is a Virus
Its structure is currently being investigated to become a vector for gene therapy
|
What is cilia
The lungs are lined with this hair-like projections that sweep the mucus microbes and debris up and out of the airways
|
What is Mitosis
What is the name of the cell division needed to aid the repair of stem cells?
|
What are Stem cells
This sort of cell was cultured by the Hubrecht Institute to demonstrate base-editing on CF patients
|
What is Cas9 enzyme
In gene editing technology, this enzyme acts as a sort of "molecular scissors" to cut and modify faulty DNA sequences
|
What is . . .
→ Persistent coughing with phlegm. → Frequent pneumonia/ bronchitis. → Male infernity. → Poor growth and weight loss. → Shortness of breath.
What is one example of the side effects of cystic fibrosis?
|
What is Symptom-management
CFTR modulators are ___-__ type of medication
|
What is Integrating Gene Therapy
This approach of treatment involves inserting DNA into cells for it to become a part of the genome
|
90%
What is the percent of people in Canada infected with cystic fibrosis who are treated with Trifaka?
|
What is Trifakta
Combines 3 drugs (ivacaftor, tezacaftor, and elexacaftor) to create a combined precision CFTR modulator
|
What are Liposomes
DNA can be packaged into a lipid coating to form this kind of vesicle for gene delivery
|
$250,000
What is the yearly cost of Trifaka in Canada, a medicated drug therapy for cystic fibrosis?
|
What is Base-editing
This technique repairs mutated genomes on-site rather than cutting and replacing with the correct codes (extension of CRISPR/CAS9 technology)
|