week 10 Flashcards
1
Q
gene therapy history of approvals
A
- 2012 - lipoprotein lipase deficiency (rare disorder) (31 people, $1M)
- 2017 - gene therapy for hereditary eye disease
- 2019 - gene therapy for spinal muscular therapy
- 2023- 5 new gene therapy approvals including first gene editing (CRISPR) approval
2
Q
Central dogma of biology
A
DNA > RNA > proteins
3
Q
gene therapy drugs
A
- gene therapies are an emerging area of drug development
- lots of clinical trials, only a few approvals
- gene therapies - when a disease involves a single gene
- change or add a healthy copy of a gene > to produce correct protein > to improve patient health
- potentially curative, but possible side effects
- costly
4
Q
case study : gene therapy for eyes
A
- disease: hereditary progressive vision loss (blindless)
- direct gene therapy to the cells of the retina
- gene: RPE65
- use a naturally-occurring viral vector
- clinical trial : 41 people
- approved by FDA in 2017
how it worked
- retina is detached from surrounding blood vessels by spraying of saline solution
- missing gene is injected into light-sensing cells of retina where it begins to produce protein and repair damaged cells
- retina reattaches within a day
5
Q
case study 2: sickle cell disease
A
- healthy individuals: red blood cell development involves stem cells
- first in human film
- hemoglobin is a protein complex made of alpha-globin and beta-globin
- sickle cell disease is one of the most common rare diseases
- remove cells, preform gene editing, deliver edited cells to patient
6
Q
Nobel prize in chemistry for CRISPR gene editing technology
A
- molecular scissors to edit human genes in 3 ways: disrupt, delete, correct/insert
7
Q
SARS-CoV-2
A
- has spike proteins that protrude out of their shell
- spike proteins bind to receptors or proteins on cells
- once inside virus releases genetic material
- infected cell reads genetic material and begins to make parts of the virus
- new copies are made and carried outside of cell
- large family of viruses that can affect human or animals
- highly transmittable
7
Q
Covid-19 vaccines
A
- several platforms have been used to develop the vaccine
- viral vector, DNA, RNA, protein, likked or attenuated virus
8
Q
viral vector
A
- modified viruses that deliver genetic code for antigen
- vectors do not contain disease-causing genes
- can be broken down into two types: replicating and non-replicating
9
Q
DNA/RNA
A
- DNA or RNA that codes for antigen protein
- can be encapsulated in a fatty coat (or bubble) to fuse with the cell membrane and deliver the genetic material inside
10
Q
protein
A
- a protein is extracted from the virus, purified, and injected as a vaccine
- mimic the coronavirus to trigger immune response
11
Q
killed or weakened virus
A
- killed virus is inactive using chemicals or heat
- weakened virus has mutations in its genome, so it doesn’t express its viral proteins as well
12
Q
why did we not find a treatment for SARS given that another viral disease is likely to reoccur
A
- after SARS outbreak, vaccines were developed
- when the virus was eradicated, the development was halted as there was no reason to revive development
13
Q
Comirnaty vaccine (BioNTech and Pfizer)
A
- mRNA that encodes the spike protein enclosed in a lipid nanoparticle (person converts mRNA to protein and mounts immune response)
- two doses administered intramuscularly 3 weeks apart
- health Canada authorized the vaccine with condition dec 9, 2020
14
Q
spikevax vaccine (Moderna)
A
- mRNA that encodes the spike protein enclosed in a lipid nanoparticle
- two doses administered intramuscularly 4 weeks apart
- health cananda authorized this vaccine with conditions on december 23, 2020
15
Q
why we got COVID-19 vaccines so quickly
A
- pandemic plans were already in place
- china identified the novel coronavirus early on
- tremendous funding was provided immediately
- speedy clinical trials process
- quick commencement of vaccine trials
- availability of research data
- studies garnered many volunteers
- rapid results from trials
- early vaccines worked well
- regulation took place while the studies were continuing
16
Q
Medicago’s plant-based COVID-19 vaccine
A
- virus-like particles are made in plants, which are then harvested and purified
- 71% efficacy against covid-19
approved feb 2022 - financial and production difficulties, was not brought to market
17
Q
drug repurposing
A
- time and cost effective strategy
18
Q
pharmacokinetic “boosting”
A
- compounds used in combination with a primary therapeutic agent that do not directly affect the disease but rather enhance/restore the activity of the primary agent
- allows for lower dosage of primary agent while maintaining therapeutic levels
- reduces pill burden and dosing frequency
- reduces extent of toxic side effects due to lower daily dose of primary drug
19
Q
long COVID
A
- symptoms persist for more than 12 months after infection
