RNA - Genetherapys

Life, Spelled Out

DNA is crucial for gene therapy as it contains the genetic instructions for the production of RNA and proteins. These genetic instructions hold the molecules for cells to function correctly and to repair genetic damage. In biomedicine, the application of DNA allows for the correction or replacement of defective genes to improve the quality of life when treating those with diseases.
Described as the DNA alphabet, the molecular structure of these genetic instructions uses 4 specific letters, A (adenine), T (thymine), C (cytosine), and G (guanine). Each forms a pair according to specific physicochemical characteristics. Life is spelled out with just four letters, how extraordinary!
Regulated by complex molecular mechanisms, every molecule within an organism, even the smallest, performs a fundamental function. The complexity of this process works like a large machine made up of many gears.
If the machine stops working properly, or if one letter is misplaced, diseases arise.

Gene Mutations: when gene therapy intervenes

Known as homeostasis, this self-regulating balance occurs as some cells duplicate, and others die in a dynamic process characterized by a steady cellular turnover. However, mistakes can occur, thus leading to mutations. This is when gene therapy intervenes in a targeted manner.
Today, the scientific community is actively involved in the search for molecular targets as a starting point for molecular modification.
There are many ways to approach gene therapy. Interventions can replace a disease-causing gene with a healthy copy of the same gene or introduce newly modified genes into the body to help treat a disease.

What are we talking about when we speak of CRISPR/Cas9?

Among the various gene-editing tools, the CRISPR/Cas9 system is widely known for its “cut-and-paste” abilities. This system allows for the identification and correction of DNA fragments by cutting non-functional parts and pasting the correct part, resulting in a functional DNA free of alterations.

Gene Therapy: two methods with a single purpose

Gene therapy represents a revolutionary breakthrough that offers innovative and effective treatments in the field of medicine. By offering a wide range of treatments that act at various levels through different mechanisms, this diversity allows us to address a broad spectrum of diseases.
Gene therapy can be given through in vivo and ex vivo methods. The in vivo method involves administering treatments directly to the patient, as in the case of CRISPR-Cas9. The ex vivo method involves the removal of cells from a patient, correcting them, and reapplying them back to the patient, as in the case of CAR-Ts. The ex vivo method using CAR-Ts consists of taking T cell lymphocytes from the patient and engineering them in the laboratory to be reintroduced, specifically attacking and destroying cancer.
The three main advantages of gene therapy
Tailor-made effectiveness: Gene therapy brings medicine towards a new era of precision and personalization, placing the patient at the center of research for specific and targeted treatments.
Single dose treatments: in most cases, a single administration is enough to obtain results, drastically reducing the need for continuous and costly treatments.
Definitive cures: this innovation represents real hope by offering definitive solutions and significantly improving the quality of life of those suffering from rare diseases.

An integration of research fields

Gene therapy requires a multidisciplinary approach.  While identifying the most suitable molecular targets, research must include the identification and development of efficient and accessible administration methods for it to succeed. An integrated synergy of skills is essential to make gene therapy an accessible reality for all patients.

Long-term investment

Although gene therapy may carry high initial costs, it offers significant long-term savings. Gene therapy eliminates the need for ongoing treatments and reduces patient care costs, representing a sustainable investment in the future of healthcare.



Written by

PharmaTech Academy student Laura De Cantis

and the Foundation’s editorial staff

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