Publisher's Synopsis
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. The concept of gene therapy arose during the 1960s and 1970s and is still in its infancy, meaning there is a paucity of reliable, long-term data on the safety and efficacy of this therapy. In 1972, Theodore Friedmann and Richard Roblin published a paper in Science called "Gene therapy for human genetic disease?" which cited Stanfield Roger's proposal in 1970 that "good DNA" could be used to replace defective DNA in people with genetic disorders. The first patient to be treated with gene therapy was a four year old girl treated at the NIH Clinical Center in 1990. She had a congenital disease called adenosine deaminase (ADA) deficiency which severely affects immunity and the ability to fight infections. Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein. A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can't cause disease when used in people. Some types of virus, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome. The vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein. This book aims to cover key aspects of existing problems in the field of development and future perspectives in gene therapy. It consists of basic and translational research, in addition to clinical experiences, and they outline functional mechanisms, predictive approaches, patient-related studies and upcoming challenges in this stimulating but also controversial field of gene therapy research. 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. 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. The concept of gene therapy arose during the 1960s and 1970s and is still in its infancy, meaning there is a paucity of reliable, long-term data on the safety and efficacy of this therapy. In 1972, Theodore Friedmann and Richard Roblin published a paper in Science called "Gene therapy for human genetic disease?" which cited Stanfield Roger's proposal in 1970 that "good DNA" could be used to replace defective DNA in people with genetic disorders. The first patient to be treated with gene therapy was a four year old girl treated at the NIH Clinical Center in 1990. She had a congenital disease called adenosine deaminase (ADA) deficiency which severely affects immunity and the ability to fight infections. Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein. A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can't cause disease when used in people. Some types of virus, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome. The vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein. This book aims to cover key aspects of existing problems in the field of development and future perspectives in gene therapy. It consists of basic and translational research, in addition to clinical experiences, and they outline functional mechanisms, predictive approaches, patient-related studies and upcoming challenges in this stimulating but also controversial field of gene therapy research. 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.
