What is Gene Therapy
Gene Therapy is an experimental technique that injects functional genes into a cell to replace missing or defective genes in order to correct genetic disorders [27]. Genes are made up of DNA. In our bodies, DNA is used to carry information needed to produce essential proteins which are the building blocks of every cell in the body [5]. When mutations occur in the human DNA strand the mutation can be harmless, but can also lead to serious diseases such as cancer, cystic fibrosis, Parkinson’s disease, cardiovascular disease, etc [5]. Gene therapy treats these diseases by repairing the dysfunctional DNA or by replacing it [5].
Researchers are currently trying to use gene therapy to inject genes into a cancer cell to catalyse the destruction of the cell or to change the cancer cells back to normal cells [5]. They are also inserting viral or bacterial DNA as a type of vaccination [5]. And to deliver genes into the body that promote tissue growth and stimulate the repair of damaged tissues [5]. Overall, scientists are working hard to discover new cures to currently incurable diseases by using gene therapy.
There are 2 main types of gene therapy [22]. One is somatic gene therapy, in which the normal DNA is inserted into somatic cells [22]. Somatic cells are cells in the body that are not germ line cells (sperm and ova), they are cells that are non reproductive [22]. In this type of gene therapy, the person’s genome is affected by the insertion of the functional DNA, but the changes are not passed on to the next generation [17]. Somatic gene therapy can also be split in to 2 forms, ex vivo and in vivo [16]. Ex vivo means exterior. In this type of gene therapy, the cells are removed from the body and injected with the virus containing the functional DNA. The cells grow in the laboratory and then are transplanted back into the body [16]. In vivo means interior. In this type of gene therapy, the cell’s gene are altered while still inside the body [16]. The picture above shows in vivo gene therapy performed [1].
Researchers are currently trying to use gene therapy to inject genes into a cancer cell to catalyse the destruction of the cell or to change the cancer cells back to normal cells [5]. They are also inserting viral or bacterial DNA as a type of vaccination [5]. And to deliver genes into the body that promote tissue growth and stimulate the repair of damaged tissues [5]. Overall, scientists are working hard to discover new cures to currently incurable diseases by using gene therapy.
There are 2 main types of gene therapy [22]. One is somatic gene therapy, in which the normal DNA is inserted into somatic cells [22]. Somatic cells are cells in the body that are not germ line cells (sperm and ova), they are cells that are non reproductive [22]. In this type of gene therapy, the person’s genome is affected by the insertion of the functional DNA, but the changes are not passed on to the next generation [17]. Somatic gene therapy can also be split in to 2 forms, ex vivo and in vivo [16]. Ex vivo means exterior. In this type of gene therapy, the cells are removed from the body and injected with the virus containing the functional DNA. The cells grow in the laboratory and then are transplanted back into the body [16]. In vivo means interior. In this type of gene therapy, the cell’s gene are altered while still inside the body [16]. The picture above shows in vivo gene therapy performed [1].
The other type of gene therapy is germ line gene therapy [17]. Germ line gene therapy inserts DNA into the person’s egg or sperm cell (reproductive cells) so that the changes are permanent and can be passed on to the offspring [17]. If germ line gene therapy is done in the early stages of embryonic development, the change in gene can occur in all cells of the developing embryo [22].
In general, the goal of gene therapy is to eliminate genetic diseases at their source [8]. With successful gene therapy, the transfer of the functional DNA will be able to correct the problem caused by the mutated DNA [8].
The picture on the right shows the process of ex vivo somatic gene therapy [25].
In general, the goal of gene therapy is to eliminate genetic diseases at their source [8]. With successful gene therapy, the transfer of the functional DNA will be able to correct the problem caused by the mutated DNA [8].
The picture on the right shows the process of ex vivo somatic gene therapy [25].