Gene Editing
AAVs are also called this
What are Adeno-associated Viruses?
CPVT or
What is Catecholaminergic Polymorphic Ventricular Tachycardia?
This is the overall result of Bezzerides et al., 2019
What is CaMKII inhibition through AAV-mediated delivery prevents arrythmias in Ryr2-mediated CPVT models?
Viral infection, drug reactions, inflammation or autoimmune reactions, and infiltrative processes are all the most common causes of this cardiomyopathy
What is nonischemic cardiomyopathy?
Researchers saw an increase in this after injecting heart failure patients with the viral vector containing the inhibitory peptide
What is Left Ventricular Ejection Fraction?
This is the method in which viral vectors are induced to their host cell
What is transduction?
Most common misdiagnosis for CPVT
What is epilepsy?
Inhibitory peptide introduced to cardiomyocytes via viral vectors targets this
What is CaMKII or calmodulin-dependent kinase II?
This class of heart failure is characterized by comfort at rest, but minor exertion leads to high level of symptoms
This phase of clinical trials involves large-scale testing
What are Phase 3 trials?
This protein regulates calcium handeling in cardiomyocytes and was targeted by researchers for inhibition
What is PP1?
60-70% of patients with CPVT have a mutation in this gene
What is Ryr2 or ryanodine receptor-2?
Result of CaMKII inhibition in human induced pluripotent stem cell derived cardiomyocytes
What is normalization of heart rhythm following adrenergic stimulation?
In failing hearts, this ion is deficient in cardiomyocytes during relaxation
What is Calcium?
The inhibitory peptide packaged onto the viral vector in Henry et al., 2025
What is AB-1002?
AAVs are specific as they do not integrate into this
What is the genome?
Two adrenergic stimuli used by researchers to induce tachycardia in mice
What are epinephrine and isoproterenol?
A common symptom of heart failure where fluid buildup causes swelling in the ankles, legs, and feet
What is edema?
This paper is an example of a certain phase of clinical trials
What is Phase 1?
This viral vector is commonly expressed in the brain and cardiomyocytes
What is AAV9?
The inheritance pattern for the most common form of CPVT
What is autosomal dominant?
Adolescent mice treated with the inhibitory peptide saw a decrease in this following adrenergic stimulation
What is induced ventricular tachycardia?
What drug for heart failure inhibits the enzyme that facilitates the conversion of angiotensin I to angiotensin II?
ACE inhibitors
The biggest limitation to the results of this study
What is a small sample size?