RNA - Genetherapys

A long name for a big mission

Many ask why the Foundation has such a long name, especially since communication channels love to use abbreviations and acronyms. So, let us discuss what is behind it!

Although we live in an age of increased digitization, some topics feel as though they are too complex and elusive for a wide audience to comprehend. Consider subjects like cancer vaccines, which can seem like an inaccessible echo of big words with an unpronounceable number of syllables. These topics feed upon the idea that science is only for the few to comprehend.

Therefore, with highly complex topics, the Foundation addresses them by engaging an entire nation on a big mission that unites its brightest and best minds.

DNA strand assembling from different elements. 3D illustration

What is the difference between RNA and DNA?

Known as the blueprint of life, Deoxyribonucleic acid, or DNA, is a long, double-stranded molecule made up of bases located in the nucleus of a cell that contains and carries our genetic code. Much like the order of letters in the alphabet form words, the order of these bases determines our genetic blueprint. The stability of the twisted double strand of DNA allows its information to continue from generation to generation, like those beautiful brown eyes you got from your mother. To read these genetic blueprints, these double strands are unraveled to expose the individual strands and an enzyme translates them into a mobile, intermediate message, called ribonucleic acid, or RNA. Unique to RNA is its ability to form pairing strands with DNA and RNA itself.

Found in complex organisms such as mammals, DNA holds its structure within a nuclear membrane called the nucleus. RNA, on the other hand, forms inside the nucleus.  However, unlike DNA, there are many types of RNA. Each type of RNA relates the functions for which they perform.  Some remain inside the nucleus and have a basic regulatory function on DNA. Others diverge into the cytoplasm, the portion outside the nucleus, and perform functions like the translation of proteins. Such proteins are the ultimate player in various tasks of the cell. They can produce membrane channels for trophic support, which control the survival and growth of the cell, and perform specific tasks like the production of membrane antigens, which represent the identity of some cells.

Why are Gene Therapy and RNA-based drugs so important?

Sometimes, cells have mutations in the gene code or within the RNAs that perform various tasks, including encoded proteins by the RNAs. These mutations result in the alteration or malfunctioning of cells that affect the proper systemic functioning of the body called homeostasis. This leads to hereditary conditions such as genetic diseases or acquired diseases such as cancer.

Now there is hope.  Scientists are tackling diseases that have been incurable for centuries, thanks to cutting-edge treatments and technology. Gene Therapy aims to correct disease-causing genetic mutations by directly inserting or modifying genes within patients’ cells. RNA-based drugs can manipulate and interfere with defective RNA or they can produce therapeutic RNAs that compensate for defective functions.

While complex, no abbreviation could do justice to the scope and ambition of our mission. Each word spells out an opportunity to revolutionize medicine and improve the lives of millions of people affected by incurable diseases thanks to the hard work and dedication of all those at the National Center who bring real hope to the field of global health.



Written by

PharmaTech Academy student Laura De Cantis

and the Foundation’s editorial staff

Latest magazine

BioPharma in pillole – RNA drugs: making sense of it all

BioPharma in pillole – Life, Spelled Out