Gene Therapy Essay

There are two main groups of gene therapy and they are germ line gene therapy and somatic gene therapy (Baksh, 2007). Germ line therapy consists of germ cells being modified by the introduction of functional genes, which are ordinarily integrated into their genomes (Garbutt et al., 2011). DNA encodes the therapeutic gene and replaces the mutated gene so the new gene can treat the disease. Germ line

Humans have been able to use the principles of DNA replication, gene transfer and gene expression (as observed in nature) as tools to manipulate specific genes towards preferred outcomes. An advantage of being able to manipulate genetic therapy is the ability to eliminate/cure genetic disease/disorders with otherwise no other cure. Humans are able to remove the genes causing the genetic disorder from the gene pool of populations, allowing organisms to live longer and healthier lives. Two ways of manipulating genetic transfer which will be explored further in this report are selective breeding and gene therapy.

One side to this issue is the use of viral vectors in gene therapy to cure the underlying disease. One of the first viral vectors used was the adenovirus vector. The adenovirus by itself is a linear double-stranded DNA molecule that causes mild respiratory infections, but when used as a vector, certain genes and regions are removed to make it less harmful (Alton, et al. 2010). The adenovirus vector is easy to grow, adaptable, able to infect both dividing and non-dividing cells and is quick to

Some people may wonder what gene therapy is. Gene therapy is the replacing of defective or missing genes with normal genes in order to cure the disorder. In the future, this technique may be used to prevent or treat a disorder by inserting

Fifty years after the idea of gene therapy was first proposed, gene therapy has become a possible treatment for a couple different diseases. Before this treatment was approved, some serious unfavorable effects were found in clinical trials. However, these effects fueled more basic research in order to improve, in efficiency and safety. Gene therapy has been used for patients with blindness, neuromuscular disease, hemophilia, immunodeficiencies, and cancer.

Gene therapy is a way in which an individual with a preexisting genetic disorder (in this case ADA deficiency) can be treated. This involves the insertion of a functioning ADA gene into an individual’s cells through the use of viral vectors, to replace the mutated ADA gene that is non-functioning or functioning incorrectly. In order for this process to be viable multiple techniques are used, including: restriction enzymes, ligation, PCR and transfection. Through these processes an individual is able to

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

In 1993, gene therapy was used to treat the infant with ADA deficiency. Immature blood cells from the babies’ umbilical cords were infused the corrected ADA genes which were reinjected into the baby.

To overcome the drawback of classic -retroviral vectors mentioned above, the authors of this study proposed the use of self-inactivating (SIN) viruses as vectors for gene therapy. A SIN retrovirus is

Somatic gene therapy is a technique of genetic therapy wherein the ‘bad’ cells are identified and examined, and specially created cells are made then implanted into the body in order to thwart the effects of the disease, counteracting their damage. This therapy is limited as only the symptoms of the disease are controlled and technically the genetic footprint of the disease is still present and if the patient were to reproduce, the offspring would still possess a chance of retaining the mutation. Somatic genetic therapy is split into two groups: ex vivo gene therapy and in vivo gene therapy. Ex vivo genetic therapy is a technique where healthy cells are extracted

As the numbers of people suffering from CNS diseases are on the rise, treatment option still remains limited for many CNS diseases. Therefore, significant unmet medical need for these diseases allows AAV Gene therapy to transform the lives of many patients. The most significant aspect of AAV gene therapy is that, it is targeted therapy which could be an effective alternative medication. Moreover, gene therapy will have a durable and meaningful benefits to the patient.

The second technique is the in situ (or "in position") therapy. Cells carrying corrective genes are introduced directly into the tissue where the genes are needed. This treatment is good for conditions that are localized, but it cannot correct systemic disorders. In situ treatment is being explored for diseases like cystic fibrosis, muscular dystrophy, and cancer. This treatment is still hindered by a lack of safe and effective ways for implanting correct genes into various organs (Anderson, 1995).

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

    Through recombinant DNA techniques, scientists have already experimented with bacteria and copied their cellular system that allows them to defend themselves from viruses in order to create a new restriction enzyme called Cas9, which then gets bound to an RNA guide strand. This new enzyme introduces new genetic material into the human genome as DNA replicates. This method will be useful for families members who carry harmful recessive alleles. Their babies could inherit the genetic disorder, but with recombinant DNA techniques, the harmful gene or genes could easily be replaced with a functional gene. Lauren Friedman, Senior editor for