0
Immune deficiencies- Several inherited immune deficiencies have been treated successfully with gene therapy. Most commonly, blood stem cells are removed from patients, and retroviruses are used to deliver working copies of the defective genes. After the genes have been delivered, the stem cells are returned to the patient. Because the cells are treated outside the patient's body, the virus will infect and transfer the gene to only the desired target cells. Severe Combined Immune Deficiency (SCID) was one of the first genetic disorders to be treated successfully with gene therapy, proving that the approach could work. However, the first clinical trials ended when the viral vector triggered leukemia (a type of blood cancer) in some patients. Since then, researchers have begun trials with new, safer viral vectors that are much less likely to cause cancer. Adenosine deaminase (ADA) deficiency is another inherited immune disorder that has been successfully treated with gene therapy. In multiple small trials, patients' blood stem cells were removed, treated with a retroviral vector to deliver a functional copy of the ADA gene, and then returned to the patients. For the majority of patients in these trials, immune function improved to the point that they no longer needed injections of ADA enzyme. Importantly, none of them developed leukemia.
Hereditary blindness-Gene therapies are being developed to treat several different types of inherited blindness-especially degenerative forms, where patients gradually lose the light-sensing cells in their eyes. Encouraging results from animal models (especially mouse, rat, and dog) show that gene therapy has the potential to slow or even reverse vision loss. The eye turns out to be a convenient compartment for gene therapy. The retina, on the inside of the eye, is both easy to access and partially protected from the immune system. And viruses can't move from the eye to other places in the body. Most gene-therapy vectors used in the eye are based on AAV (adeno-associated virus). In one small trial of patients with a form of degenerative blindness called LCA (Leber congenital amaurosis), gene therapy greatly improved vision for at least a few years. However, the treatment did not stop the retina from continuing to degenerate. In another trial, 6 out of 9 patients with the degenerative disease choroideremia had improved vision after a virus was used to deliver a functional REP1 gene.
Hemophilia-People with hemophilia are missing proteins that help their blood form clots. Those with the most-severe forms of the disease can lose large amounts of blood through internal bleeding or even a minor cut. In a small trial, researchers successfully used an adeno-associated viral vector to deliver a gene for Factor IX, the missing clotting protein, to liver cells. After treatment, most of the patients made at least some Factor IX, and they had fewer bleeding incidents.
TO SEE MORE GO TO: http://learn.genetics.utah.edu/content/genetherapy/gtsuccess/
Still curious? Ask our experts.
Chat with our AI personalities
Rene
Jordan
EzraSuccessful gene delivery in gene therapy trials is characterized by high efficiency, specificity, and safety. Efficient delivery ensures a high percentage of target cells receive the gene of interest. Specificity ensures that the gene is delivered only to the intended target cells. Safety involves minimal off-target effects, immune responses, and long-term stability of gene expression.
Add your answer:
Earn +20 pts
When will be gene therapy successful?
Gene therapy is already successful for certain genetic disorders, such as SMA and beta-thalassemia, with ongoing research to expand its applications. The field is rapidly evolving, and as technology advances and more clinical trials are conducted, we can expect gene therapy to become increasingly successful in treating a wider range of diseases in the near future.
In the SCID-X1 gene therapy trials what problem later surfaced in three of the patients?
In the SCID-X1 gene therapy trials, three patients developed leukemia as a result of the treatment. This was due to the unexpected activation of an oncogene during the insertion of the corrective gene into the patients' cells.
What are some of the possible benefits of using magnet therapy?
Magnet therapy allegedly promotes healing and decreases pain, possibly by affecting the flow of blood. However, these claims have not been verified in formal randomized controlled trials. Strong magnets, like those found in MRI machines, have little effect on ordinary humans; those used in magnet therapy are much weaker.
Is cystic fibrosis a candidate good for gene therapy?
Yes, cystic fibrosis is a candidate for gene therapy. Gene therapy offers the potential to correct the underlying genetic cause of the disease, which is a mutation in the CFTR gene. While there have been some promising developments in gene therapy for cystic fibrosis, further research and clinical trials are needed to fully assess its effectiveness.
Where can I find information on stem cell therapy?
One of the most important scientific and medical advances in India in recent years has been stem cell therapy. The nation is now firmly established and well-positioned in the global sphere of medical science in the arena of stem cell therapy. You can find more about stem cell therapy at advancells.
