Gene therapy for inherited diseases
Order ID |
53563633773 |
Type |
Essay |
Writer Level |
Masters |
Style |
APA |
Sources/References |
4 |
Perfect Number of Pages to Order |
5-10 Pages |
Description/Paper Instructions
Gene therapy for inherited diseases
Gene therapy is an innovative approach to treating inherited diseases that involves altering the genetic makeup of an individual. Inherited diseases are caused by mutations or changes in a person’s genes that are passed down from their parents. Gene therapy seeks to correct these mutations or introduce new genes to replace or supplement the mutated ones. In this article, we will discuss the benefits of gene therapy for inherited diseases and its potential for revolutionizing healthcare.
One of the primary benefits of gene therapy for inherited diseases is that it offers a potential cure for diseases that were previously untreatable. Inherited diseases can cause significant morbidity and mortality, and traditional treatments often only provide temporary relief of symptoms. Gene therapy, on the other hand, seeks to address the underlying genetic cause of the disease and potentially provide a permanent cure. This can improve the quality of life for patients and reduce the burden on healthcare systems.
Gene therapy also has the potential to treat a wide range of inherited diseases, from rare genetic disorders to more common conditions such as cystic fibrosis and sickle cell disease. By targeting the specific genetic mutations that cause these diseases, gene therapy can provide a more targeted and effective treatment approach than traditional therapies.
Another benefit of gene therapy for inherited diseases is that it can be administered early in life, potentially preventing the onset of symptoms or slowing disease progression. This is particularly important for diseases that have a high morbidity and mortality rate in early childhood, such as spinal muscular atrophy and Huntington’s disease.
One of the challenges of gene therapy for inherited diseases is the complexity of the genetic makeup of an individual. Gene therapy requires a thorough understanding of the specific genetic mutations that cause the disease, as well as the delivery of the correct gene to the appropriate cells in the body. Additionally, gene therapy can have potential risks, such as off-target effects or immune reactions.
Despite these challenges, there have been several successful gene therapy trials for inherited diseases. For example, Luxturna, a gene therapy approved by the FDA in 2017, is used to treat inherited retinal dystrophy, a rare genetic disorder that causes vision loss. The therapy works by delivering a functional copy of the gene responsible for producing a protein that is essential for vision to the retina.
Another successful gene therapy is Zolgensma, approved in 2019 for the treatment of spinal muscular atrophy. This disease affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. Zolgensma works by delivering a functional copy of the SMN1 gene, which is responsible for producing a protein necessary for motor neuron survival.
In conclusion, gene therapy offers a promising treatment approach for inherited diseases by targeting the underlying genetic mutations that cause these conditions. While there are still challenges to overcome in the development and delivery of gene therapies, the potential benefits for patients are significant. Gene therapy has the potential to revolutionize healthcare by providing a more targeted and effective treatment approach for inherited diseases, potentially leading to cures for previously untreatable conditions.
RUBRIC
QUALITY OF RESPONSE |
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POOR / UNSATISFACTORY |
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EXCELLENT |
Content (worth a maximum of 50% of the total points) |
Zero points: Student failed to submit the final paper. |
20 points out of 50: The essay illustrates poor understanding of the relevant material by failing to address or incorrectly addressing the relevant content; failing to identify or inaccurately explaining/defining key concepts/ideas; ignoring or incorrectly explaining key points/claims and the reasoning behind them; and/or incorrectly or inappropriately using terminology; and elements of the response are lacking. |
30 points out of 50: The essay illustrates a rudimentary understanding of the relevant material by mentioning but not full explaining the relevant content; identifying some of the key concepts/ideas though failing to fully or accurately explain many of them; using terminology, though sometimes inaccurately or inappropriately; and/or incorporating some key claims/points but failing to explain the reasoning behind them or doing so inaccurately. Elements of the required response may also be lacking. |
40 points out of 50: The essay illustrates solid understanding of the relevant material by correctly addressing most of the relevant content; identifying and explaining most of the key concepts/ideas; using correct terminology; explaining the reasoning behind most of the key points/claims; and/or where necessary or useful, substantiating some points with accurate examples. The answer is complete. |
50 points: The essay illustrates exemplary understanding of the relevant material by thoroughly and correctly addressing the relevant content; identifying and explaining all of the key concepts/ideas; using correct terminology explaining the reasoning behind key points/claims and substantiating, as necessary/useful, points with several accurate and illuminating examples. No aspects of the required answer are missing. |
Use of Sources (worth a maximum of 20% of the total points). |
Zero points: Student failed to include citations and/or references. Or the student failed to submit a final paper. |
5 out 20 points: Sources are seldom cited to support statements and/or format of citations are not recognizable as APA 6th Edition format. There are major errors in the formation of the references and citations. And/or there is a major reliance on highly questionable. The Student fails to provide an adequate synthesis of research collected for the paper. |
10 out 20 points: References to scholarly sources are occasionally given; many statements seem unsubstantiated. Frequent errors in APA 6th Edition format, leaving the reader confused about the source of the information. There are significant errors of the formation in the references and citations. And/or there is a significant use of highly questionable sources. |
15 out 20 points: Credible Scholarly sources are used effectively support claims and are, for the most part, clear and fairly represented. APA 6th Edition is used with only a few minor errors. There are minor errors in reference and/or citations. And/or there is some use of questionable sources. |
20 points: Credible scholarly sources are used to give compelling evidence to support claims and are clearly and fairly represented. APA 6th Edition format is used accurately and consistently. The student uses above the maximum required references in the development of the assignment. |
Grammar (worth maximum of 20% of total points) |
Zero points: Student failed to submit the final paper. |
5 points out of 20: The paper does not communicate ideas/points clearly due to inappropriate use of terminology and vague language; thoughts and sentences are disjointed or incomprehensible; organization lacking; and/or numerous grammatical, spelling/punctuation errors |
10 points out 20: The paper is often unclear and difficult to follow due to some inappropriate terminology and/or vague language; ideas may be fragmented, wandering and/or repetitive; poor organization; and/or some grammatical, spelling, punctuation errors |
15 points out of 20: The paper is mostly clear as a result of appropriate use of terminology and minimal vagueness; no tangents and no repetition; fairly good organization; almost perfect grammar, spelling, punctuation, and word usage. |
20 points: The paper is clear, concise, and a pleasure to read as a result of appropriate and precise use of terminology; total coherence of thoughts and presentation and logical organization; and the essay is error free. |
Structure of the Paper (worth 10% of total points) |
Zero points: Student failed to submit the final paper. |
3 points out of 10: Student needs to develop better formatting skills. The paper omits significant structural elements required for and APA 6th edition paper. Formatting of the paper has major flaws. The paper does not conform to APA 6th edition requirements whatsoever. |
5 points out of 10: Appearance of final paper demonstrates the student’s limited ability to format the paper. There are significant errors in formatting and/or the total omission of major components of an APA 6th edition paper. They can include the omission of the cover page, abstract, and page numbers. Additionally the page has major formatting issues with spacing or paragraph formation. Font size might not conform to size requirements. The student also significantly writes too large or too short of and paper |
7 points out of 10: Research paper presents an above-average use of formatting skills. The paper has slight errors within the paper. This can include small errors or omissions with the cover page, abstract, page number, and headers. There could be also slight formatting issues with the document spacing or the font Additionally the paper might slightly exceed or undershoot the specific number of required written pages for the assignment. |
10 points: Student provides a high-caliber, formatted paper. This includes an APA 6th edition cover page, abstract, page number, headers and is double spaced in 12’ Times Roman Font. Additionally, the paper conforms to the specific number of required written pages and neither goes over or under the specified length of the paper. |
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Gene therapy for inherited diseases
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