Gene therapy is a medical procedure that replaces or corrects genetic defects. It can be used to treat a large variety of diseases, including cancers, metabolic disorders and blood disorders. Gene therapy has been studied in clinical trials for more than 30 years, but it hasn’t yet become widely available due to safety concerns about using the most common type of vector for this approach: viruses. Say’s Dr Francene Gayle ,however, new advances are making it possible for this type of treatment to become more widely available as early as 2022.
Researchers have been working for nearly three decades to develop gene therapies for people with rare diseases.
In the past 30 years, researchers have been working to develop gene therapies for people with rare diseases. The first gene therapy trial was in 1990 and many of the trials have been on cancer patients. There are many different types of rare diseases, including ones that affect your skin or immune system. Some rare conditions can be passed down through families, while others occur when something goes wrong during development before you’re born (called congenital).
Gene therapy involves replacing a mutated or missing gene with a normal one.
Gene therapy involves replacing a mutated or missing gene with a normal one. Genes are the basic units of heredity, and they are made up of DNA that’s organized into chromosomes in the nucleus of cells. DNA is composed of four types of nucleotides: adenine (A), cytosine (C), guanine (G) and thymine (T).
The replacement gene must be inserted into the patient’s cells in such a way that it will be expressed properly; otherwise, it may have no effect on their condition or even make things worse if there is an adverse reaction from introducing foreign material into the body.
There are several forms of gene therapy, including viral vector and DNA-based approaches.
- There are several forms of gene therapy, including viral vector and DNA-based approaches.
Viral vectors are viruses that have been modified to carry genes into a patient’s cells. They can be used to treat any condition caused by a single defective gene or multiple mutations in different genes. Viral vectors work by inserting their genetic cargo into the nucleus of the cell where it integrates into DNA and begins producing proteins needed for normal function. This process is called transduction because it takes place across generations (trans) within your body’s cells (duct). Some common examples of viral vectors include adeno-associated virus (AAV) and lentiviruses such as HIV-1/2 (which causes AIDS).
DNA-based approaches involve directly injecting DNA into the body where it will integrate into existing chromosomes in order to express its encoded protein(s).
The most commonly used vectors for this approach are adeno-associated virus (AAV) or lentivirus.
The most commonly used vectors for this approach are adeno-associated virus (AAV) or lentivirus. Both are small viruses that don’t cause disease, but can be engineered to deliver genes to the liver in clinical trials.
Gene therapy is an exciting area of research with potential to help people with rare diseases. We are still in the early stages of development, but we are making great strides towards a cure for some of these diseases.