What is the treatment of genetic diseases with replacement genes called?
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updated: October 19, 2022 DefinitionGene therapy is a technique that uses a gene(s) to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in a patient’s cells with a healthy version of that gene. Both inherited genetic diseases (e.g., hemophilia and sickle cell disease) and acquired disorders (e.g., leukemia) have been treated with gene therapy.
 NarrationGene therapy. Gene therapy is a direct way to treat genetic conditions as well as other conditions. There are also other related approaches like gene editing. There are many different versions and approaches to gene therapy and gene editing. It all rests on understanding how genes work and how changes in genes can affect our health. Researchers all over the world are studying many different facets of gene therapy and gene editing. SearchGene therapy is currently an experimental treatment that replaces or inactivates disease-causing germs through injection or IV. Research continues into the ways this therapy can treat cancer and other conditions.
OverviewWhat is gene therapy?Gene therapy is an experimental treatment using genetic material to treat or prevent certain diseases. While not yet widely available, gene therapy may one day help doctors treat diseases by directly replacing the disease-causing gene. Clinical trials are investigating gene therapy for the treatment of cancer, age-related macular degeneration and other eye diseases, certain genetic conditions and HIV/AIDS. Currently, one gene therapy medication, Luxturna®, has been approved by the U.S. Food and Drug Administration (FDA) for use in the United States. Luxturna treats certain inherited retinal (eye) diseases. Procedure DetailsHow does gene therapy work?Gene therapy works by replacing or inactivating disease-causing genes. In some cases, gene therapy introduces new genes into the body to treat a specific disease. With gene therapy, doctors deliver a healthy copy of a gene to cells inside the body. This healthy gene may replace a damaged (mutated) gene, inactivate a mutated gene or introduce an entirely new gene. Carriers, called vectors, transport these healthy genes into cells. In most cases, the vectors are modified viruses that do not cause disease. Vectors may also be certain types of bacteria or circular DNA molecules (plasmid DNA). Additional methods to package and deliver genetic material are also being actively investigated, such as the use of nanoparticles, encapsulating lipid molecules and the use of electric currents. Injection or intravenous (IV) infusion introduces vectors into the body. In some cases, doctors collect cells from a patient, add vectors in a laboratory and return the vector-containing cells to the patient’s body through injection or IV infusion. Risks / BenefitsWhat are the risks and benefits of gene therapy?With the exception of Luxturna which has been FDA approved, doctors are still experimenting with gene therapy. The long-term safety of such treatments has yet to be determined. Some gene therapies appear to be effective in curing certain conditions. But there is not enough evidence about gene therapy as a whole to determine all the possible risks. Some gene therapy research indicates gene therapy may worsen symptoms or cause them to last longer. Additionally, complications of certain gene therapies may include cancer, toxicity and inflammation. Recovery and OutlookWhat is the outlook after gene therapy?Your recovery depends on which medical condition gene therapy treats. Complications can be serious and can affect your outcome. When to Call the DoctorWhen should I ask my doctor about gene therapy?Researchers are investigating gene therapy to treat cancer, eye diseases, some genetic conditions and HIV/AIDS. If you are interested in participating in a clinical trial involving gene therapy, speak with your doctor. Your doctor can help determine whether gene therapy might treat your condition. Get useful, helpful and relevant health + wellness information
enews More health news + infoWhat is the new gene therapy called?A newer technique, called genome editing (an example of which is CRISPR-Cas9), uses a different approach to correct genetic differences. Instead of introducing new genetic material into cells, genome editing introduces molecular tools to change the existing DNA in the cell.
What are the 4 types of gene therapy?The Technologies. Vectors for Gene Transfer. Vectors used for gene therapy include modified versions of natural viruses and plasmids. ... . In Vivo Gene Therapy. In vivo gene therapy involves direct injection of the gene therapy agent into the body. ... . Ex Vivo Gene Therapy. ... . Gene Editing. ... . Chimeric Antigen Receptor T-Cell Therapy.. What are the 3 types of gene therapy?There are basically three types of gene therapy: ex vivo, in vivo, and in situ. In ex vivo gene therapy, the target cells are removed from the patient's body, engineered either by the addition of the therapeutic gene or by other genetic manipulations that allow correction of the phenotype of the disease.
What are the treatment of genetic?The only known treatment for the variety of genetic diseases sometimes called "inborn errors of metabolism," or "storage diseases," is a bone marrow transplant (BMT).
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