Gene therapy
"Although it's early days for these trials, the approach of modifying a patient's T-cells to attack their cancer is looking increasingly like one that will, in time, have a place alongside more traditional treatments," says Paul Moss of Cancer Research UK.
Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:
Replacing a mutated gene that causes disease with a healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
Introducing a new gene into the body to help fight a disease.
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures.
For general information about gene therapy:
MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapy.
The fact sheet Gene Therapy from the U.S. Department of Energy Office of Science offers an overview of this topic.
Educational resources related to gene therapy are available from GeneEd.
The Genetic Science Learning Center at the University of Utah provides an interactive introduction to gene therapy.
The Centre for Genetics Education provides an introduction to gene therapy, including a discussion of ethical and safety considerations.
Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Scientific breakthroughs continue to move gene therapy toward mainstream medicine.
Scientists first took the logical step of trying to introduce genes directly into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular dystrophy and sickle cell anemia. However, this has proven more difficult than modifying bacteria, primarily because of the problems involved in carrying large sections of DNA and delivering them to the correct site on the comparatively large genome. Today, most gene therapy studies are aimed at cancer and hereditary diseases linked to a genetic defect. Antisense therapy is not strictly a form of gene therapy, but is a related, genetically-mediated therapy.
Gene therapy using an adenovirus vector: A new gene is injected into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.
The biology of human gene therapy remains complex and many techniques need further development. Many diseases and their strict genetic link need to be understood more fully before gene therapy can be used appropriately. The public policy debate surrounding the possible use of genetically engineered material in human subjects has been equally complex. Major participants in the debate have come from the fields of biology, government, law, medicine, philosophy, politics, and religion, each bringing different views to the discussion.
There are a variety of different methods to replace or repair the genes targeted in gene therapy.
A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common.
An abnormal gene could be swapped for a normal gene through homologous recombination.
The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function.
The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.'''
Spindle transfer is used to replace entire mitochondria that carry defective mitochondrial DNA
Further Reading
Gene Therapy Types
Gene Therapy Vectors
Gene Therapy History
Gene Therapy Issues
This article is licensed under the Creative Commons Attribution-ShareAlike License. It uses material from the Wikipedia article on "Gene therapy" All material adapted used from Wikipedia is available under the terms of the Creative Commons Attribution-ShareAlike License. Wikipedia® itself is a registered trademark of the Wikimedia Foundation, Inc.
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"Although it's early days for these trials, the approach of modifying a patient's T-cells to attack their cancer is looking increasingly like one that will, in time, have a place alongside more traditional treatments," says Paul Moss of Cancer Research UK.
Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:
Replacing a mutated gene that causes disease with a healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
Introducing a new gene into the body to help fight a disease.
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures.
For general information about gene therapy:MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapy.
The fact sheet Gene Therapy from the U.S. Department of Energy Office of Science offers an overview of this topic.
Educational resources related to gene therapy are available from GeneEd.
The Genetic Science Learning Center at the University of Utah provides an interactive introduction to gene therapy.
The Centre for Genetics Education provides an introduction to gene therapy, including a discussion of ethical and safety considerations.
Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Scientific breakthroughs continue to move gene therapy toward mainstream medicine.
Scientists first took the logical step of trying to introduce genes directly into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular dystrophy and sickle cell anemia. However, this has proven more difficult than modifying bacteria, primarily because of the problems involved in carrying large sections of DNA and delivering them to the correct site on the comparatively large genome. Today, most gene therapy studies are aimed at cancer and hereditary diseases linked to a genetic defect. Antisense therapy is not strictly a form of gene therapy, but is a related, genetically-mediated therapy.Gene therapy using an adenovirus vector: A new gene is injected into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.
The biology of human gene therapy remains complex and many techniques need further development. Many diseases and their strict genetic link need to be understood more fully before gene therapy can be used appropriately. The public policy debate surrounding the possible use of genetically engineered material in human subjects has been equally complex. Major participants in the debate have come from the fields of biology, government, law, medicine, philosophy, politics, and religion, each bringing different views to the discussion.
There are a variety of different methods to replace or repair the genes targeted in gene therapy.
A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common.
An abnormal gene could be swapped for a normal gene through homologous recombination.
The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function.
The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.'''
Spindle transfer is used to replace entire mitochondria that carry defective mitochondrial DNA
Further Reading
Gene Therapy Types
Gene Therapy Vectors
Gene Therapy History
Gene Therapy Issues
This article is licensed under the Creative Commons Attribution-ShareAlike License. It uses material from the Wikipedia article on "Gene therapy" All material adapted used from Wikipedia is available under the terms of the Creative Commons Attribution-ShareAlike License. Wikipedia® itself is a registered trademark of the Wikimedia Foundation, Inc.
Recent Gene Therapy News
Study: Gene therapy can boost heart muscle function
In an animal study, researchers at the University of Washington show that it is possible to use gene therapy to boost heart muscle function. The finding suggests that it might be possible to use this ...
Study raises alarm over loss of individual 'genomic liberty' due to gene patents
Humans don't "own" their own genes, the cellular chemicsals that define who they are and what diseases they might be at risk for. Through more than 40,000 patents on DNA molecules, companies have esse...
Novel cell therapy effective in treating aggressive form of childhood leukemia
Two children with an aggressive form of childhood leukemia had a complete remission of their disease—showing no evidence of cancer cells in their bodies—after treatment with a novel cell therapy that ...
Two studies shed light on molecular biology of three blood disorders
Two studies led by investigators at Weill Cornell Medical College shed light on the molecular biology of three blood disorders, leading to novel strategies to treat these diseases.
Researchers expand hematopoietic stem cells for bone marrow transplantation
More than 50,000 stem cell transplants are performed each year worldwide. A research team led by Weill Cornell Medical College investigators may have solved a major issue of expanding adult hematopoie...
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