StudentShare
Contact Us
Sign In / Sign Up for FREE
Search
Go to advanced search...
Free

Gene Therapy - Research Paper Example

Cite this document
Summary
Gene Therapy: Problems and Solutions to Problems Gene therapy is a method of treating various acquired and genetic diseases with genes. This is an experimental technique and is done by inserting genetic material into the patient’s cells. In this form of treatment, the diseases are either treated or prevented by replacing abnormal gene, or by introducing the absent gene or by supplementing the defective gene (Genetics Home Reference, 2008)…
Download full paper File format: .doc, available for editing
GRAB THE BEST PAPER96.5% of users find it useful
Gene Therapy
Read Text Preview

Extract of sample "Gene Therapy"

Gene Therapy: Problems and Solutions to Problems Gene therapy is a method of treating various acquired and genetic diseases with genes. This is an experimental technique and is done by inserting genetic material into the patient’s cells. In this form of treatment, the diseases are either treated or prevented by replacing abnormal gene, or by introducing the absent gene or by supplementing the defective gene (Genetics Home Reference, 2008). The genes thus introduced exert their benefits by either compensating for the abnormal genes or by producing beneficial protein or by supplementing the abnormal gene (Hecht, n. p.).

The first gene therapy clinical trial started in 1990 (Verma and Weitzman, n. p.). Currently, research is going on for gene therapy by various approaches in different parts of the world for various diseases like combined immune deficiencies, hemophilia, Parkinson's disease, cancer and even HIV (Verma and Weitzman, n. p.). Though gene therapy has been under research for the past 2 decades, no single gene therapy has been approved for clinical use. The main reason for this is safety concerns pertaining to gene therapy trials and treatment.

In this essay, problems associated with gene therapy and strategies to prevent those problems will be reviewed. Safety issues related to gene therapy are mainly related to the methods employed in gene therapy. There are basically 2 methods of gene therapy and they are ex-vivo therapy and in-vivo therapy. In ex-vivo therapy, gene delivery is done in cells after being removed from the body (Hecht, n. p.). The cells used are basically grown in the laboratory. The cells are than modified outside the body and then transplanted back into the body.

In some research trials, cells from blood or born marrow are taken out and cultured in a laboratory. Thereafter, the cells are exposed to the virus with the desired gene. The virus infects the cells and transfers the therapeutic genetic material into the nucleus of the cells. After this, the cells are injected into the patient’s body by vein. In vivo therapy, gene delivery is being done in the cells that are still in the body. The genetic material can be delivered by different routes. The specific route is determined by the disease.

Gene therapy for cystic fibrosis is administered by inhalation. In hemophilia and cancer treatment, the gene is directly injected into the tumor (Hecht, n. p.). Viral vectors can introduce toxicity, immune and inflammatory responses. Also, though the viruses are engineered to be innocuous, there is always a fear that they may cause disease (Hecht, n. p.). Immunogenicity is an important factor with regard to safety of the patient subjected to gene therapy trial. The immunogenicity is that of the vector and it is derived from the proteins of the virus and the capability of the viruses to infect macrophages and dendrite cells.

Such an immune response can result in elimination of the transduced cells and also the vector (Hecht, n. p.). The potency and dose are very important aspects for good outcomes in gene therapy and these are basically linked to the production process and also the purification process (Raty et al., n. p.). These processes maximize the infective virus and decrease the number of defective viruses or empty viral particles. Using administration methods to increase the concentration of the virus locally helps in decreasing the side effects and also aids in lowering the amount of total dose that is administered into the patient.

In the ex-vivo approach, cells of the patient that are outside the body like skin can be used for transduction for augmentation of the grafting of the tissue and also for providing therapeutic protein secretion. These methods help in limiting the exposure to the patient to vectors of gene delivery, thus increasing safety aspects. These methods are however, laborious. Another method of decreasing the amount of dose administered to the person is by combining lipoproteins of low density with avidin (Raty et al., n. p.). In this method, substances that are bitinylated are captured to the cells from the circulation, thus effectively increasing the drug concentration locally.

This helps in reducing the side effects by limiting the amount of drug concentration in other tissues. Certain tissue specific promoters when used with gene therapy vectors along with promoters that can be regulated can help in decreasing the amount of vectors exposed to the body. Another strategy is to delete the replication genes in the vector, mostly adenovirus, so that the viruses are gutless. Currently third generation vectors are being used which are much safer. Improvement in chromatographic methods have decreased the purification of viruses and also the delivery of reservoirs like silicon collars or collagen collars which decrease systemic leakage of viruses along with tissue specific promoters.

Imaging the bio-distribution of particles of virus can help in gene therapy specificity, thus improving safety aspects (Raty et al., n. p.). To summarize and conclude, introduction of genetic material into the cells for therapeutic purposes is known as gene therapy. This mode of treatment is useful for diseases with gene absence, mutation or insufficiency. However this is still in experimental stage. Gene therapy offers hope to those with incurable genetic disorders, but is fraught with several challenges and side effects.

Improvement in the chromatographic methods has enhanced the purification of viruses and also the delivery reservoirs. Other strategies which can reduce harm due to gene therapy viruses are using of tissue specific promoters and also imaging of the viral bio-distribution. Works Cited Hecht, F. “Gene Therapy - The Future Is Here!” MedicineNet.com. 2004. Web. 10th May, 2013 . Raty, J.K., H. P. Lesch, T. Wirth, and S. Yla-Hertuala. “Improving Safety of Gene Therapy.” Current Drug Safety, 3, 46-53. 2008. Print.

Genetics Home Reference What is Gene Therapy? 2008. Web. 10th May, 2013 . Verma, I.M., Weitzman, M.D. “Gene Therapy: Twenty-first Century Medicine.” Annu Rev Biochem, 74, 711-38. 2005. Print.

Read More
Tags
Cite this document
  • APA
  • MLA
  • CHICAGO
(“Gene Therapy Research Paper Example | Topics and Well Written Essays - 750 words”, n.d.)
Gene Therapy Research Paper Example | Topics and Well Written Essays - 750 words. Retrieved from https://studentshare.org/biology/1477905-gene-therapy
(Gene Therapy Research Paper Example | Topics and Well Written Essays - 750 Words)
Gene Therapy Research Paper Example | Topics and Well Written Essays - 750 Words. https://studentshare.org/biology/1477905-gene-therapy.
“Gene Therapy Research Paper Example | Topics and Well Written Essays - 750 Words”, n.d. https://studentshare.org/biology/1477905-gene-therapy.
  • Cited: 0 times

CHECK THESE SAMPLES OF Gene Therapy

Aspects on How Gene Therapy Works

Instructor Institution Date Gene Therapy Introduction Gene Therapy is the therapeutic use of genes as medication.... Gene Therapy involves the transfer of a working copy or therapeutic genes into specific cells of a person with an aim of repairing a gen copy that is faulty (Fackelmann 239).... hellip; It is important to note that Gene Therapy is still an experimental approach to therapy.... The history of Gene Therapy and the related concepts can be traced to the 1960s and early in the 1970s....
8 Pages (2000 words) Research Paper

Ethics of Gene Therapy

Gene Therapy remains one such debated issue with maximum ethical criticisms despite the wide spectrum of benefits it offers to mankind. Gene Therapy is a concept derived from cloning domain which defined as the "replication of one or more individual plants or animals, (whole or in parts) through asexual scientific method creating exact copies of the parent cell"....
5 Pages (1250 words) Case Study

What is Gene Therapy

The author of the essay under the title "What is Gene Therapy" states that using genes to prevent or treat a disease is known as Gene Therapy (NIH, 2008).... The first Gene Therapy clinical trial started in 1990 (Hecht, 2004).... Currently, research is going on for Gene Therapy by various approaches.... The uses of Gene Therapy are to replace absent or defective genes, deliver genetic material into cells that speed the destruction of cancer cells, deliver genetic material into cells that cause cancer cells to revert back to normal cells, deliver genes of bacteria or virus as a form of vaccination, deliver genetic material that either impedes or promotes growth of a new tissue and deliver genetic material that stimulates healing of a damaged tissue (Hecht, 2004)....
1 Pages (250 words) Essay

Practical Considerations in Gene Therapy

… Yet, today Gene Therapy is considered for cases where risks, especially risks to life, are not even involved.... Using the perspective of Culver (1994a), Cotrim and Baum explained that Gene Therapy is a procedure that “typically involves the insertion of a functioning gene cells to correct dysfunction or to provide new cellular functions.... ?? Culver (1994a) is a physician's handbook on Gene Therapy.... In 1994, Culver (1994b) had already outlined a procedure for the clinical applications of Gene Therapy for cancers....
8 Pages (2000 words) Essay

The use of gene therapy to treat genetic diseases

In each individual, there are two copies of beta globin hemoglobin An individual has sickle cell trait when one of the two copies is carrying the sickle cell beta globin gene.... On the other hand, if both the copies carry sickle cell beta globin gene then the individual has sickle cell anemia and suffers from the debilitating disease.... An individual with normal beta globin hemoglobin genes has children with an individual possessing two copies of sickle cell gene have a 100% probability that all their children will have sickle cell trait i....
4 Pages (1000 words) Essay

GENE THERAPY (VIRAL AND NON-VIRAL VECTORS)

AAV happens to be unique among the viruses under development for Gene Therapy due to HVS (Harpes Simplex Virus Vector) happens to be the most complex as well as the largest among all viruses under development for Gene Therapy with one of its features of importance being that it has a capacity that is able to carry he fragments for foreign DNA (Mohammed, Al-Dosari1, & Xiang 2009).... Several applications of Gene Therapy look promising in clinical early phase trial for instance haemophilia B treatment by use of rAAV, vascular and coronary artery disease treatment by use of viral vectors and certain kinds of cancer treatment by use of “conditionally replicating ecolytic viruses” (Kenneth & Teni 2003)....
3 Pages (750 words) Essay

Fetal Gene Therapy

The paper "Fetal Gene Therapy" describes that generally, Mattar CN has conducted various researches with other scientists on various pediatric issues and other healthcare issues.... The only setback in prenatal Gene Therapy is concerns about its safety, efficacy, regulatory and ethical issues.... In the article also, the authors have evaluated current practices in prenatal Gene Therapy and potential success in future clinical practice.... he authors of this article have covered the topic on fetal Gene Therapy exhaustively; hence, his article is a noteworthy source for anyone wishing to enhance knowledge about the topic....
5 Pages (1250 words) Annotated Bibliography

Ethical Considerations in Gene Therapy

This paper "Ethical Considerations in Gene Therapy" attempts to investigate the various unsolved ethical problems associated with Gene Therapy treatment techniques.... nbsp;Since the concept of Gene Therapy cannot be considered as a novel variation or recent advance, the history of ethical issues associated with Gene Therapy dates back to the time when the term was first suggested.... Gene Therapy technique is applied to develop efficient methods for the introduction of DNA into cells after appropriate DNA sequences and cell types have been identified....
7 Pages (1750 words) Essay
sponsored ads
We use cookies to create the best experience for you. Keep on browsing if you are OK with that, or find out how to manage cookies.
Contact Us