In the past, diseases caused by genetic defects had little to no way of being cured. Most treatments focused on managing the problems, rather then solving them. However, not more then a few decades ago, research began to surface for what would become a procedure that focused on adresing the root of the problem, the gene.
The Beginnings
It all started with simple speculations. The first scientific paper regarding looking at the problem from the perspective of the gene was published in 1980, by David Williams, MD, and David Nathan, MD, and it proposed the use of a virus for the insertion of genes in blood-developing stem cells. The following years, research lead to the creation of gene therapy, a new form of therapy targeting genetic defects.
Variations of Gene Therapy
There are two main types of gene therapy: Ex vivo and In vivo. Ex vivo operates trough removing cells from the patient, in order to introduce new genetic material, packaged in a delivery vehicle called a vector. In vivo works by injecting the IV infusion of the vector directly in to the bloodstream or in the target organ. This therapy is also divided between somatic gene therapy, the proces that focuses on replacing or preventing a faulty gene that was either hereditary or developed due to environmental factors, and germline gene therapy, the proces that focuses on preventing such a defect by operating on the egg or the sperm cells.
Different Procedures
There are a lot of proceses that are tied on to the label of gene therapy, because the field itself is very vast and still in a developing phase. Some genetic diseases may require bone marrow transplants, in wich a persons cells get removed from the bone marrow, in order for them to be modified and returned. The viral vector is a virus that has been modified in a way that prevents it from causing infection, so it can be safely inserted in to the body with the correct DNA or RNA sequence. Stem cells can also be used for this type of therapy, also trough genetic modification then insertion. Liposomes, another gene therapy tehnique, uses fat to deliver the genetic material. Making disease cells aparent to the immune system is also a proces that falls in to the category of gene therapy.
Risks of Gene Therapy
This seemingly world changing tehnique doesnt come without risks, tough. Gene therapy has the potential to cause an unwanted immune system reaction, infections due to the viral vector and, in an even more unfortunate case, tumors that can lead to cancer. There is also a posibility that the wrong cell will be afected by the treatment.
Who Can Benefit
The ones that can benefit from this type of treatment are people suffering from Cystic fibrosis, Sickle cell disease, Lebers hereditary optic neuropathy (LHON), retinal disease and many others, including some types of cancer.
Conclusion
The medical potential that this still-in-the-works method of addresing several genetic diseases has is inmesurable. Even if the field of gene editing is a largely controversal one, gene therapy might be the answer millions of people affected by genetic diseases are looking for. Editing DNA and RNA sequencing can also prevent such problems from apearing in the future, saving many from the pain that these diseases could of brought.
No responses yet