Gene therapy for respiratory 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 respiratory diseases
Gene therapy is an emerging treatment modality for respiratory diseases that aim to correct or replace faulty genes with functional ones. It is a promising approach for the treatment of a wide range of respiratory conditions, including cystic fibrosis, alpha-1 antitrypsin deficiency, and pulmonary arterial hypertension.
Cystic fibrosis is a genetic disorder that affects the lungs and other organs, leading to recurrent infections and difficulty breathing. Gene therapy for cystic fibrosis aims to correct the underlying genetic defect that causes the disease, either by replacing the faulty gene with a functional one or by providing a functional copy of the gene. A recent example of gene therapy for cystic fibrosis is the use of viral vectors to deliver a functional copy of the CFTR gene to the lungs. In clinical trials, this approach has shown promising results, with patients experiencing significant improvements in lung function and a reduction in respiratory infections.
Alpha-1 antitrypsin deficiency is another respiratory disease that can be treated with gene therapy. This condition is caused by a deficiency of alpha-1 antitrypsin, a protein that protects the lungs from damage. Gene therapy for alpha-1 antitrypsin deficiency aims to provide a functional copy of the alpha-1 antitrypsin gene to the lungs, either through viral vectors or other gene delivery methods. This approach has shown promise in preclinical studies and is currently being tested in clinical trials.
Pulmonary arterial hypertension is a condition in which the blood pressure in the lungs’ arteries is elevated, leading to difficulty breathing and an increased risk of heart failure. Gene therapy for pulmonary arterial hypertension aims to correct the underlying genetic defects that cause the disease, either by replacing the faulty gene with a functional one or by providing a functional copy of the gene. This approach is still in the early stages of development and is currently being tested in animal models and early-phase clinical trials.
In conclusion, gene therapy holds great promise for the treatment of respiratory diseases. While the field is still in its early stages, ongoing research and clinical trials are yielding promising results and bringing us closer to realizing the full potential of gene therapy for the treatment of respiratory diseases. However, there are still several challenges that need to be overcome, including the development of safe and effective gene delivery methods, the identification of appropriate candidate genes, and the development of appropriate animal models for the study of these diseases. Nevertheless, the future of gene therapy for respiratory diseases is bright, and the potential benefits for patients are enormous.
RUBRIC
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POOR / UNSATISFACTORY |
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GOOD |
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 respiratory diseases
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