Group 5-2A:

Parimah Rajabi Eslami

Donya Rezvani

Sabri Altan Sepetcioğlu

Zahra Seifi

Danial Dadfar

mRNA based Therapy

(Noble prize winner subject)
 What is mRNA?
mRNA, or messenger ribonucleic acid, is a macromolecule within cells that works to
synthesize protein from genes encoded in an organism’s DNA. mRNA is transcribed from
DNA within a cell’s nucleus.

Our DNA, or genetic code, contains all the genes we will express in our lifetime. Within these
genes are instructions that code for different proteins. Our cell’s everyday function is
dependent on the availability of specific proteins.

mRNA is the “middleman” between our cell’s DNA and proteins. “Messenger RNA ” earns its
name as it carries DNA’s genetic code instructions to make a specific protein. The use of
mRNA in drug therapeutics have had a revolutionary effect on treating various diseases.
mRNA therapies are versatile and programmable in nature and recognizable to the body
through its natural synthesis of mRNA.

The Function of mRNA in Body Cells

Once DNA transcribes into mRNA, the mRNA travels to the cytoplasm and attaches to a
ribosome, where protein synthesis takes place.

Proteins are responsible for nearly every task within cells.

When mRNA is transcribed from DNA, our genetic code can then be processed into proteins
that can carry out the DNA’s instructions for gene expression.

These proteins can provide structural support for our cells and orchestrate their everyday
functions.
How Does mRNA Therapy Work?
mRNA-based therapy involves delivering sequences of mRNA into the body. This is achieved
by encapsulating the mRNA into a delivery vehicle, such as liposomes or nanoparticles. An
example of mRNA therapy is the COVID-19 vaccine, which utilizes lipid nanoparticles (LNPs).

The LNPs are loaded with mRNA sequences that synthesize COVID-19 spike proteins found
on the virus’s surface. When the LNPs bind to and enter cells, the mRNA is released. The
body then reads these mRNA sequences and synthesizes the COVID-19 spike protein. The
immune systems response to the spike protein result in antibody production that helps
prevent the virus from attacking healthy cells

Why is mRNA Therapy a Good Alternative to Current Treatments?

mRNA therapy has shown to be a versatile treatment method for a range of diseases due to
its programmable nature. mRNA therapies can utilize targeted drug delivery to reach

specific cells within the body while having little to no effect on surrounding areas.

Depending on the mRNA therapy’s design, various proteins can be coded for by a given
sequence.

Additionally, mRNA is an incredibly stable molecule allowing easy storage and transportation.
Its use also poses limited side effects since it is a naturally occurring molecule produced
within the human body.

Nevertheless, mRNA therapies are breakthrough treatment methods. Much development and
research are still needed to make these therapies widely available and ensure their efficacy.

mRNA Treatment Design

1. Insertion of mRNA into DNA Plasmid
The protein of interest must be identified and isolated when designing mRNA therapeutics.
Once this occurs, the protein coding sequence can be inserted into an artificial DNA segment,
such as a plasmid.

2. Transcription of DNA into mRNA

This DNA segment is then transcribed into mRNA. This mRNA will, which will, in turn, code
for the protein of interest.

3. Loading mRNA into Delivery Vehicles and Binding and Entry into
Target Cells
Once the mRNA segment is isolated, it can be loaded into a delivery vehicle such as
liposomes or lipid-based nanoparticles. It is crucial to maintain the stability of the mRNA while
it is enveloped within the delivery vehicle to ensure successful delivery to target cells.

4. Protein Synthesis
The mRNA is released when the delivery vehicles bind to and enter cells. Once in the body,
the ribosomes of the target cells read the mRNA, and the synthesis of the protein of interest
begins.

Types of mRNA Therapies

Due to mRNA’s versatile and stable nature, a range of mRNA therapies exist to treat
diseases. Treatments include vaccinations and therapies that target protein availability and
immune responses.

mRNA Vaccines

mRNA Protein Replacement Therapy

mRNA Immunotherapy and Implications for Cancer Treatment

The Future of mRNA Therapies and
FDA Approval
mRNA therapies are promising avenues for treating various diseases due to their versatile
and programmable nature. Nevertheless, many mRNA technologies are still in early
development, such as the use of mRNA in gene therapies.

The use of mRNA in the COVID-19 vaccine has seen success in preventing the disease and
paved a successful future for mRNA therapies. Since the breakthrough of the COVID-19
vaccine, FDA has established fast tracks to making life-saving drugs readily available to the
public, which only strengthens the future of mRNA therapies.

mRNA Treatment Drawbacks

mRNA therapies are breakthrough treatments still in early development. Specific limitations
include:

• The cost of mRNA therapy development.

• Stability of the mRNA molecule. • The stability of the
mRNA carrier.

Cost of mRNA Therapy Development

Stability of mRNA Molecules

Stability of Average mRNA Carriers