GENE THERAPY FOR HAEMATOLOGICAL DISORDERS: Gene therapy can be defined as the use of DNA obtained through genetic engineering as a therapeutic pharmaceutical agent in treatment diseases. In other words it is an experimental techniques whereby the absent or faulty gene is replaced by a working gene, so that the body can make the correct enzyme or protein and consequently eliminate the root cause of the disease.
Gene therapy aims to correct the disease process by restoring, modifying or enhancing cellular functions through introduction of a functional gene into target cell. Whilst the concept of gene therapy is simple, the practical reality of translating this new technology to the clinic has proven to be more difficult than first imagined.
Recent progress in gene transfer technology has shown impressive clinical success in some diseases, however, there was development of leukaemia in some patients and even death which in previous trails caused a major setback to the development of this technology.
This reviews tends to outline the meaning of gene therapy, types, methods, application in haematological and other disorders, certain obstacles to its full implementation and possible ways put forward to tackle these problems facing development of gene therapy.
The word “therapy” literally means “curing or healing” and is the attempted remediation of a health usually following a diagnosis in the medical. There are many therapies that have been developed for various diseases since ages, these include:
- Chemotherapy: this deals with treatment of cancer using one or more cytotoxic antineoplastic drugs as part of a standardized regime. It is often used in conjunction with other cancer treatment such as radiation or surgery. However ,certain chemotherapeutic agents act by killing cells divide rapidly(cancer cells) which is also harmful to normal rapid dividing cells as bone marrow, digestive tract cells and hair follicles, thus resulting to most common side effects such as ;myelosuppression, mucositis and alopecia. Chemotherapy is being improved nowadays using targeted therapy –a form of classic chemotherapy.
- Radiotherapy: this is the medical use of ionizing radiation generally as part of cancer treatment to control or kill malignant cells.It may be curative in a number of types of cancer if they are localized to one area of the body, although with its own side effect, this has been used for treatment of cancer for more than 100years.
- Surgery: deals with removal of part of the diseased cells or tissues. It is an invasive procedure.
- Acupuncture: this is a collection of therapeutic procedures that deals with the stimulation of points on the body using a variety of techniques, such as penetrating the skin with needles that are then manipulated manually or by electrical stimulation. It is one of the key components of traditional Chinese medicine (TCM) and is among the oldest healing practices in the world (en.wikipedia.com).
Current research suggests that traditional forms of acupuncture are more effective than placebos in the relief of certain type of pain and post operative nausea (Lee and Fan, 2009.). Recent systematic reviews found that acupuncture also seems to be a promising treatment option for anxiety, sleep, disturbance and depression but that further research is needed in these regards (Lee, et al,2012).
Acupuncture’s use for certain conditions has been endorsed in some countries such as America and Untied Kingdom because there is general agreement that acupuncture is relatively safe when administered by qualified practitioners using sterile needles and carries a very low risk of series adverse effects (White, 2004)
- Homeopathy is a system of medicine which involves treating the individuals with highly diluted substances, given mainly in tablet form, with the aim of triggering the body’s natural system of healing. It was discovered by a German doctor, Samuel Hahoe mann, whose aims was to reduce the damaging side effects of associated with medicine treatment then such as blood-letting, purging and the use of poisons such as arsenic.
Homeopathy is based on the principle that in healthy people “like cures like” i.e a substance which causes symptom when taken in large doses can be used in small amount to cure those same symptoms in sick people. Example, excess intake of coffee causes sleeplessness and agitation thus according to this principle, when made into a homeopathic medicine, it could be used to treat people with these symptoms.
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The scientific community regards homeopathy as sham, quakery and it remedies have been criticized as unethical based on the fact that its principle says that the potency of a drug increases with low dilution and not the other way round.
- Gene therapy: This is the most recent form of therapy in medicine that deals with the use of invitro coded genes as a therapeutic agent in treatment of diseases.
The compelling desire to target therapy at a molecular level has resulted in the emergence of new direction in medicine called gene therapy.
Gene therapy can be defined as the use of DNA as a therapeutic pharmaceutical agent in treatment of diseases. The term derives its name from the idea that DNA can be used to supplement or alter genes within an individuals cell as a therapy to treat disease. Gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene i.e. modifying and enhancing cellular functions through the introduction of a functional gene into a target cell.
A gene is a molecular unit of heredity of a living organism. It can be a DNA or RNA that codes for a polypeptide or for an RNA chain that has a function in the organism. Every individual inherits a copy of gene from both parents, and a particular gene inherited has its own biological function thus any mutation affecting the gene that codes for a particular protein will definitely result in a genetic disease. Genetic diseases both inherited and acquired have posed a wide challenge to the scientific community.
Since 1970s, the scientific and ethical issue arising out of proposals for genetic manipulation in humans were already widely discussed with a general conclusion that genes therapy for severe disorders for which there was no corrective treatment would be ethically acceptable and analogues to the use of drugs to treat specific diseases (Anderson, 1972; Fruedman and Robin, 1972).
Gene therapy being a technique for correcting defective gene responsible for diseases development, researchers have derived several approaches for correcting faulty genes, these include;
- Replacing a mutated gene that causes diseases with a healthy copy of the gene.
- Inactivating or knocking out a mutated that is functioning improperly.
- Introducing a new gene into the body to help fight a disease.
- The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.