September 20

Gene therapy: meet the medicine of the future

United Kingdom of Great Britain and Northern Ireland flag icon
Reading time 4 minutes

In the XX century, medicine and pharmacy made an incredible leap. A wide variety of medicines were created and put into widespread practice — from antibiotics to the first therapeutic antibodies — thanks to which the health and well-being of many people significantly improved, as well as the average life expectancy increased. However, progress cannot be stopped: the delivery of the necessary genes directly into the cells and tissues of the body or their directed editing allows you to "fix" faulty molecular processes, which gives fundamentally new opportunities for the treatment of previously incurable diseases in comparison with traditional pharmaceuticals. And since technologies do not stand still, in the future, gene therapy will take an important place in the arsenal of doctors. In this article, we will focus specifically on gene therapy, analyze how it differs (and how it is similar) to cell therapy, consider the technologies used, go through the currently existing drugs, and also touch on the future prospects and existing limitations of such treatment.

The principle of operation: how gene therapy works

To begin with, let's understand the terminology, and for this we will consider the definition of gene therapy introduced by one of the world's main regulators of the pharmaceutical field — the US Food and Drug Administration (FDA). So, according to the FDA, GENE THERAPY is a medical intervention based on the modification of the genetic material of living cells. The cells can be modified ex vivo for subsequent administration to humans or modified in vivo directly in the body.

How does it work?

To begin with, it is worth saying that gene therapy today consists mainly in transferring certain genes to human somatic cells, since changing the genome of germ line cells for therapeutic or other purposes is prohibited in most countries with appropriate technologies. Therefore, changes in genetic information during such treatment are not inherited and affect mainly only certain target cells in the body.

Gene therapy is also classified depending on whether changes are made to cells directly in the body or outside it. In vivo gene therapy involves the introduction of genetic material directly by infusion; in this case, a solution containing a certain number of necessary genes, usually enclosed in carriers (or vectors — more about them below), is injected into the human body. After that, the introduced genetic constructs reach the target cells and, getting into them, are expressed there in the corresponding protein products.

In contrast, in ex vivo gene therapy, the necessary cells are first " withdrawn "from the patient, and the genetic material is injected into them in vitro (that is, in the laboratory), after which such cells are" multiplied " in a Petri dish to a sufficient amount and injected back to the patient. It is obvious that ex vivo gene therapy is also a cell therapy, since it uses the transplantation of genetically modified cells into the human body.

Carrier vectors for gene delivery

In most cases, viruses are used as carriers for gene delivery, whose natural ability to introduce their genetic material into the host cells can be not only harmful, but also useful. But everything is not as scary as it seems: such viruses are first "dissected" by genetic engineering methods, removing most of the genes responsible for virulence, which does not allow the virus to divide uncontrollably and frees up space for inserting the target gene that needs to be delivered to the cell. These carrier viruses do everything the same as before, but only for the benefit of the patient's health: they protect the therapeutic gene from cleavage by blood enzymes, force target cells to capture the virus, separate nucleic acid from the viral particle and transport it to its destination (usually to the cell nucleus). A cell that has "caught" such a virus turns out to be infected ― but not with a dangerous infection, but with a gene that treats the disease ― and therefore begins to synthesize a new protein that guides the cell along the path of healing.

Gene therapy today

Today, we are closer than ever to entering a new era of gene therapy, because many drugs of this class for use for various indications are already at different stages of clinical trials (of which more than two thousand have been registered). Nevertheless, since gene therapy initially encountered serious obstacles and failures on the way to introduction into the clinic, as well as due to the complexity of development, very few such drugs have been approved for the actual treatment of patients so far.

A look into the future

Gene therapy using viral vectors will certainly continue to improve, and we will see new drugs based on this technology in the clinic. However, there are solutions that theoretically can enhance the potential of gene therapy, which, perhaps, in the future will make it not only much safer and more effective, but also applicable to more common diseases (however, while such achievements are far away, we will not get too far ahead).

We are talking about point editing of the genome, which received a second life in 2012 with the development of the CRISPR/Cas9 tool — a technology originating from the natural system of protecting bacteria from viruses (bacteriophages), but adapted by researchers to their needs, and now a popular tool for genetic manipulation. This method is relatively easy to use, has a fairly high accuracy, and therefore has managed to find application in a variety of fundamental research, including experiments on cell lines, laboratory animals, plants, and now in clinical trials of gene therapy based on it.

Limitations of gene therapy

Gene therapy technologies are at the very beginning of the path, so, naturally, they have a lot of" childhood diseases " and a huge scope for improvement. The main tasks that need to be solved are listed below:

  1. complexity, labor intensity, high cost and, as a result, poor scalability of the technology, which is why gene drugs are still incredibly far from mass production (and it's not a fact that they will approach it soon);
  2. the exorbitant cost of such treatment resulting from the previous paragraph, which is why it is available only to a few;
  3. often serious adverse events of a new type, sometimes even leading to irreversible consequences (up to a fatal outcome). However, as they gain experience, doctors are already learning to cope with them;
  4. insufficient observation period for the use of gene therapy, hence there is uncertainty about the long-term effect of its effect, as well as concerns about the possible long-term consequences of the use of such drugs;
  5. Imperfection of the target gene delivery technology: the viral vectors that are most often used are far from ideal. In addition to the mentioned problems with non-targeted integration into the genome (characteristic of lentiviral and gamma-retroviral vectors), there is also a danger of activation of the immune system (for AAV vectors), which reduces the efficiency of delivery of target genes. At the same time, the repeated introduction of the vector is not always possible.

However, these are not only problems: these are also potential "growth points" for a new technology, where new ideas get a chance to be realized in the form of a tangible advantage.

Comments

Leave your questions here

To write a comment you must
or
Services
Category filter
Concern filter
Type filter
Sort
 
All categories
Art Therapy
$50 USD
Artist Teacher Workshop Or Tutoring

“Life Force” guiding our history, our body and spirits. Self portrait art, life drawing from the live model, gesture, and plein air painting. And extended cont…

Bryan Prillwitz
United States of America flag icon
Tarot Reading
$100 USD
Timeline Reading

EGYPTIAN TAROT READINGS using unique methods developed by Nelise Carbonare, a Brazilian healer with more than 40 years of experience in tarot consultations, as…

Nelise Carbonare
United States of America flag icon
Tarot Reading
$120 USD
THE SYNERGY OF THE PYRAMID

THE SYNERGY OF THE PYRAMID focuses on a specific relationship: One side for the consultant and one for their partner. (the partner does not need to be present …

Nelise Carbonare
United States of America flag icon
Tarot Reading
$10 USD
Pick one card

Many questions can be answer with just one Arcane.

Do you want to try?

Nelise Carbonare
United States of America flag icon
Personal Development Coaching
$50 USD
Spiritual Coaching - Removing Energy Blocks - Expansion

> “Who looks outside dreams. Who looks inside awakens.” Jung

Spiritual coaching is about moving you into a profound state of empowerment and involution m…

Kimla Rose (Kim Desrosiers)
United States of America flag icon
Life Coaching
$10 USD
Life Purpose and Soul Wisdom

What is your deeper truth, your deeper knowing, your MOJO, your deepest desires, those feelings that you can’t explain but that are so pure and so clear…or may…

Anna Beale
United States of America flag icon
Wellness Coaching
$10 USD
Your Missing Manual for Health & Life!

Are you constantly reading books, blogs and listening to podcasts trying to figure out what to do to be healthy?

With so much conflicting information out ther…

Pamela Malo
United States of America flag icon
Nutritional Therapy
$150 USD
Nutritional Therapy Consultation

Hi my name is Aria and I am the founder of Heal with Nutrition. I am a registered Nutritional Therapist and a Naturopath who has a passion on Nutrition and Hea…

Aria Alexandrou
United Kingdom of Great Britain and Northern Ireland flag icon
Tarot Reading
$10 USD
THOTH AND HERMETIC DECKS READING

With a deep knowledge of hermeticism and being introduced to the kabbalah tradition of the emanation process from the tree of life, I provide a deep insight co…

James Marchiori
Ireland flag icon
Personal Development Coaching
$55 USD
The Bridge to Your Desired Life

This is were you get real help with your struggles and life challenges. As life gets harder to cope with, we tend to look for external ways to get through. And…

Mina Mikhail
United States of America flag icon
Naturopathy
$10 USD
Intake Consult

short introduction consult to Integrative Medicine

Femke Neervoort
Netherlands flag icon
Career Coaching
$300 USD
Reset Coaching and Mentoring Programme

12 weeks of bi-weekly 90 minutes 1:1 Coaching and Mentoring. Total cost USD1800 (6x90minutes career coaching and mentoring session)

Are you returning to work …

Carla Martins
United Kingdom of Great Britain and Northern Ireland flag icon
Personal Development Coaching
$120 USD
Spiritual Life Coach Session-Personalized Session

Personalized Sessions

Because Everyone Is Unique

Client-centered sessions based on the area of focus requested by the client.

Each session is a LIVE Sessio…

Deborah Lucero
United States of America flag icon
Reiki
$55 USD
Guidance, Inspiration & Healing

This type of session is a favorite among many of my clients, as it is often times one of the smoothest way to address a challenge to reach a happier and health…

Mina Mikhail
United States of America flag icon
Mediumism
$10 USD
Psychic Readings

I specialize in love, past present future, future, past life, general, tarot, dream interpretation, specific, social, and career readings! Book today for clari…

Holly
United States of America flag icon
Life Coaching
$399 USD
1-on-1 Coaching

The journey started with our discovery call, and You decided: ” I’m ready! Let’s do this!”.

We will engage in 1-on-1 coaching sessions for 90 minutes. In this…

Carla Martins
United Kingdom of Great Britain and Northern Ireland flag icon
Ascension
$88 USD
Ascension Mentor Support

In this session, we come together with similar to a healing session but face to face.

We talk about what issues you are facing at the moment and we delve deep…

Claire C.
Australia flag icon
Spiritual Healing
$177 USD
Energy Healing

I am honored to assist you with intuitive channeled energy healings that support and nurture you on a physical level as well soul and energetic level, each ses…

Claire C.
Australia flag icon
Reiki
$375 USD
Usui Reiki 1st Degree Course

FIRST DEGREE COURSE - The ultimate Reiki course.

Have you ever wondered if you can heal? Have you felt a burning desire to help others? Then you’ve come to th…

Step Into Your Light - Christina Moore
United Kingdom of Great Britain and Northern Ireland flag icon
Life Coaching
$10 USD
Hand Analysis - Startup Session

What does a hand analysis do for me? ​

You will understand yourself and your life purpose. ​You will understand the roots of your triggers and reactionary be…

Brent Bruning, Master Hand Analyst
Switzerland flag icon
Shamanism
$10 USD
Sample Akashic Records Reading for Your Burning Issue

I connect to your Akashic Records and listen to what your guides want to bring to your conscoius attention at this time. Perhaps, it is an insight into why you…

Olga Aydınoğlu
Turkey flag icon
Shamanic Healing
$300 USD
Shamanic Healing Art

What is Shamanic Healing Art?

It is a physical artwork (usually a pastel drawing) I make for you based on the outcome of our online session. This artwork will…

Olga Aydınoğlu
Turkey flag icon
Shamanic Healing
$70 USD
Heal to say "No"

A limits and boundaries shamanic healing session to help you heal relevant wounds and traumas from this and/or other lifetimes, let go of false beliefs, judgem…

Olga Aydınoğlu
Turkey flag icon
Shamanic Healing
$70 USD
Writer's Block Shamanic Healing Session

Before the Session

I do my pre-meditation to find out the main reasons for your writer’s block. (This happens psychically, where I generally just need your na…

Olga Aydınoğlu
5
Turkey flag icon