Which disorder has been treated by in vivo gene therapy?
Table of Contents
In this review, we cover both preclinical animal studies and clinical human trials that have used ex vivo gene therapy to treat neurological disorders with a focus on Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, ALS, and stroke.
What is an example of ex vivo gene therapy?
The four most common viral vectors used for both ex vivo and in vivo gene therapy are adenovirus, adeno-associated virus (AAV), lentivirus, and retrovirus.
What is in situ gene therapy?
In situ gene therapy consists of the administration of the gene product to a specific site. It possesses several advantages, such as the reduction in potential side effects, the need for a lower vector dose, and, as a consequence, reduced costs, compared to intravenous administration.
When is ex vivo gene therapy used?
Selecting an ex vivo treatment may be better suited for treating conditions that affect the blood, such as hemophilia, where a person’s own self-renewing stem cells can be engineered with a functional working gene and then delivered into their body.
What are the benefits of in vivo gene therapy?
In vivo gene therapy benefits from its simplicity and the reduced volume of material introduced into the brain (Freeman, 1997). These properties make it an ideal method when the target is a specific, small area of the brain.
Which of the following is most controversial approach in gene therapy?
This approach is known as germline gene therapy. The idea of these germline alterations is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known.
Which type of chromosomal mutation will lead to Alagille syndrome?
Alagille syndrome is related to the JAG1 gene mutation. It may be passed from parent to child. If you have one parent with Alagille syndrome, you have a 50% chance of developing the condition. It appears in one out of 70,000 babies and occurs in both sexes.
Does gene therapy change DNA?
Gene therapy involves altering the genes inside your body’s cells in an effort to treat or stop disease. Genes contain your DNA — the code that controls much of your body’s form and function, from making you grow taller to regulating your body systems. Genes that don’t work properly can cause disease.
How is ex vivo gene therapy done?
Ex vivo gene therapy refers to the process of removing specific cells from a person, genetically altering them in a laboratory, and then transplanting them back into the person. Learn more about VECTORS and how they deliver genetic material in gene therapy.
What are the benefits of in vivo therapy?