Using Modified Dna Inserted From An Organism With A Missing Or Mutated Sequence?

Gene therapy is a very controversial topic that has been discussed in the last five years and is being heavily studied to help cure cancer. Gene therapy is a technique aimed at treating genetic disorders by introducing the correct form of the defective gene into the patient’s genome (Dunlop et al., 2010).

DNA has about 12 million single nucleotide polymorphisms and thousands of copy number variants, most of which are not harmful. Sometimes these mutations in genes can cause genetic disorders that end up altering or inhibiting protein function. Gene Therapy focuses on correcting the mutated genes usually by inserting a normal gene into the genome. Researchers treat these diseases this way by repairing dysfunctional gene or by providing copies of the missing gene. They isolate the normal DNA and package it into a vector. Other ways include replacing the abnormal gene with a normal one, repair the mutated gene, or altering the regulation of a certain gene. As of right now, reparative results have been marginal, there is potential of gene therapy treating single-gene disorders.

There are two types of gene therapy; germline gene and somatic gene therapy. Germline gene therapy is considered to be a safer option for humans. This method makes changes to the gamete cells that are used in the reproductive process thus, the functional genes need to be inserted into the chromosomes. Somatic gene therapy is when genes are transferred to body cells by inserting vectors into a person’s body. There are three things that gene therapy can do; replace a mutated gene that causes a disease with a healthy gene, deactivate a mutated gene that is not able to function properly, or introduce a new gene into the body to prevent diseases. There has been a clinical trial in the past where over 3000 people were treated with gene therapy and the results were good however, this treatment method has serious health

Li, and Huang discuss the controversial topic of gene therapy by first providing a factual summary of gene therapy. Their introduction goes into the specifics of the types of vectors (carriers that deliver a gene to target cells) used to transfer genes in order to aide damaged proteins. Afterwards, they transition into the detailed of process of gene therapy. They start this off with naked DNA and state it is the simplest approach to nonviral transfers and the least toxic method. However, despite being the least toxic and a nonviral transfer it does not have as great of an effect as Cationic lipids. Cationic lipids have been used in various experiments but are known to be safe only when administered in small doses. Furthermore they go into

Genetic disorders have plagued people for ages. However, recent research and new discoveries in gene therapy has brought new hope to those unfortunate individuals that are ailed by their own genetics. Gene therapy is a technique of correcting defective gene sequences specifically responsible for the disease. In 1990, the first successful clinical trial of gene therapy was initiated for adenosine deaminase deficiency (GT). Since then, the number of clinical treatments induced worldwide has increased exponentially. Gene therapies have been a part of modern medicine for almost two decades now. Only recently have scientists started to deliver on their immense promise after a string of setbacks raised doubts about the safety of manipulating human DNA. The idea of gene therapy has been around for a while, but it wasn’t until recently that this field of molecular biology has come more into the light with its advancements over time. Experimentation has been an ongoing process with gene therapy. Although, ethical issues of controversy have surrounded this topic since the beginning. But new facts on gene therapy continues to rebuttal most controversy.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), but There are going to be many mistakes. What are we going to do with the mistakes?

The objective of gene replacement therapy is to alter the defective gene to achieve a permanent cure for the diagnosed patient by identifying the faulty gene and providing a corrected copy of the gene without affecting the genomic sequence. Clinical trials initiated gene replacement experimentation, primarily leading to significant progress but not yet accomplished.“The most recent clinical trial for the treatment of hemophilia with gene transfer showed transient achievement of efficacy in the highest dose cohort tested, but also exposed a previously hidden barrier to the future success of these treatments.” (Gene Therapy for Haemophilia) Recently, the focus of gene therapy strategies is mostly on gene addition rather than gene replacement.

In gene therapy, mediums are need to transfer the correct genes into the body. In order to be a suitable vector, they must be able to keep the genes safe from degeneration, to transport the gene into the cell in question, and to establish secure gene transcription when the genes enter the nucleus.1 They also need to be safe for the patient, and have none to minimal risk of infection or other sort of danger to the patient. As of this year, more than 40 different mediums have been approved, thus are safe to use; these are divided up into viral and nonviral

According to Blau and Springer (1995), gene therapy is a newly rising approach at the end of the 20th century and aims to open new door for the treatment of various human diseases based on transfer of genetic materials to cells. The recent gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy is not inherited and thus it cannot be passed down on to a person’s children in the future. Meanwhile, gene therapy could also be targeted to egg and sperm cells (knowns as germline gene therapy) which would allow the inserted gene to be passed on to the progeny. Germline gene therapy allows the correction of disease-causing

This technology can target specific diseases on genes such as Alzheimer’s, Parkinson’s, polio, cystic fibrosis and the list can go on forever (Hackett, 2013). A popular method used to implement new genes is the adenovirus. A gene cannot simply be integrated into cells, it must have a vector, or a carrier which in this case is the adenovirus which helps integrate the gene into the DNA of the targeted cells (NLM, 2015). Another option is available where the subject’s cells are removed and injected with the gene and reinserted into the body (NLM, 2015). Both methods have shown success. Recently, in Oxford retinal disease has been overcome by using gene therapy (McClements, 2013). Several cases in this study allowed men and women to overcome a range of retinal diseases and avoid blindness and this is all due to the amazing effects of gene therapy (McClements, 2013). They used the adenovirus as a vector to deliver the new genes into the retinal cells in order to make the change in the eye. It was a hard fought success. There are several cases just like this study that have allowed people to renew their lives through human gene

Gene therapy pro-actively transfers new genes into a patient therefore treating or preventing the disease. Gene therapy protocols follow two paths. Somatic gene therapy is a process in which hybrid retro vector is inserted into the cell. The vector binds to the cell membrane, packages itself in the vesicle which breaks down releasing it into the nucleus. Retroviruses are vectors harnessed for this task because their RNA genetic information can easily be transcribed into DNA. Furthermore, retroviruses introduce enzymes like integrase into the cell. Integrase incorporates new genes into the genome of a host. However, integrase can insert the modified genetic material in any arbitrary position in the genome of the host leading to insertional mutagenesis, and possibly cancer (Gene Therapy Retrovirus). Alternatively, cloned DNA solution is physically injected into a solution of zygote pronuclei during germline gene therapy In this way, the zygote produced has modified DNA which is passed to future generations. Nevertheless, germline therapy has a low rate of transgene integration in which modified genes do not get incorporated into the genome of the zygotes (Smith Kevin

Gene therapy is an experimental treatment, which is used to replace mutated, improperly functioning genes with regular DNA. There are several gene therapy methods, most of which involve using a vector to transport the corrected gene into targeted cells. Retro viruses, adeno viruses, adeno ascosciated viruses and liposomes are commonly used as

Gene therapy is an experimental technique that allows doctors to insert a gene into a patient’s cell rather than using drugs or surgery. Gene therapy is a process of which defective or undesired genes in the body with “normal” genes. A vector is re-engineered to deliver the gene to a target cell. Then the gene is transferred to the cell’s nucleus and must be activated in order to function. The main focus of gene therapy is to replace a lost or improper gene with a new functional copy into a vector that is inserted into the subject’s genome by way of penetrating its DNA. Gene therapy can be done outside of the body known as ex vivo by way of taking cells form patients bone marrow or blood and then growing them

Gene therapy was introduced in 1985 by SyTech and has been the main priority of the company ever since due to its promising future in science. One of the first human gene therapy procedures involved injecting the correct ADA gene to a four year old girl with the ADA deficiency. Due to the success of the procedure, the patient is now living a healthy life. The success marked an important landmark in human history, one that has the potential to change the future.

disease (NLM, 2014). The procedure uses a corrected gene and inserts it into a person instead of