Babson College Societal Implications of CRISPR Cas9 Paper

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Research Paper: • • • Write an 8-10-page research paper on a chemistry or biochemistry topic and its larger impact on society. This work should demonstrate your ability to apply the knowledge and skills gained in your overall learning experience to a deeper understanding of a complex question(s), problem(s), or challenge(s) from multiple perspectives. Title page: The title page should include the following: a. A brief, descriptive title that reflects the experiment being reported. b. Your name c. The course name and number. Body: This section should be 8-10 pages in length. It should be organized to contain the following. - A clear introduction that introduces a chemistry or biochemistry topic and its application or impact on society. - A body that explains the chemistry or biochemistry concept in detail, and explores questions, problems, and challenges of how the topic applies in a larger societal context looking through multiple perspectives (ex. social, economic, ethics, etc.) - Should contain at least one infographic, one table, and 1-2 images that are meaningful and strengthen delivery of the subject matter. Images and tables should be no more than three inches in height. • Conclusion: The paper should have a clear conclusion section that provides a summary of everything that has transpired since the introduction. It should reveal the relationship between different points made throughout the paper. It also is an opportunity to re-examine your topic statements and explore future directions, solutions, or personal perspectives to the topic discussed in the paper. • References: Should contain at least 7-10 references correctly cited throughout the paper using the ACS citation format outlined in the ACS style guide. General guidelines 1. The paper should be typed using either 12 pt. Times New Roman or 12 pt. Arial font and doublespaced. 1” margins all around. 2. All pages should be numbered. 3. Provide citations for reference source for any literature information you provide. Citations should be numbered and should be indicated as a superscript number or in parenthesis. 4. Figures should be self-contained, labeled appropriately, and embedded within the body of the text. They should be numbered sequentially and include a figure legend below the figure that describes what is shown (e.g. Fig. 1. Description…). Similarly, tables should have a table legend (e.g. Table 1. Description…) Title: Societal Implications of CRISPR-Cas9 as Treatment for Duchenne Muscular Dystrophy Thesis: CRISPR-Cas9 has potential implications on society, but it is useful as a treatment for Duchenne muscular dystrophy and its optimization should be pursued. I. Introduction A. CRISPR-Cas9 B. DMD C. DMD Treatment with CRISPR II. Pros/Cons of CRISPR-Cas9 as DMD Treatment A. Uses/Strengths/Benefits B. Risks/Weaknesses/Drawbacks III. Ethics A. Moral Aspect of CRISPR-Cas9 as DMD Treatment B. Concerns/Any harm? IV. Financial Cost A. Any effects on economy V. Conclusion A. Summarize B. Long-Term Effects C. How could this shape society for the future? Annotated References • Conboy, Irina et al. “Making gene editing a therapeutic reality.” F1000Research vol. 7 F1000 Faculty Rev-1970. 21 Dec. 2018, doi:10.12688/f1000research.16106.1 This review article summarizes challenges with genome editing using CRISPRCas9 for medical applications and recent attempts to overcome these obstacles in clinical trials for the medical use of CRISPR-Cas9. It also discusses the details of gene editing to treat dominant and recessive genetic myopathies like Duchenne muscular dystrophy and myotonic dystrophies. • Rossant, Janet. “Gene editing in human development: ethical concerns and practical applications.” Development (Cambridge, England) vol. 145,16 dev150888. 25 Jul. 2018, doi:10.1242/dev.150888 This article highlights the use of gene editing, such as CRISPR-Cas9 and related techniques, for research in primates, human embryos, and stem cells, along with ethical issues with potential germline genetic editing. Even though technical difficulties and safety concerns prevent the practical use of these methods in clinical applications, the new information from this research about human development and the origins of pluripotent stem cells, among other things, is also discussed. • Kc, Mandip, and Clifford John Steer. “A new era of gene editing for the treatment of human diseases.” Swiss medical weekly vol. 149 w20021. 27 Jan. 2019, doi:10.4414/smw.2019.20021 In this review article, it is explained how gene editing is revolutionizing the treatment and possible cure of certain diseases. It also elaborates upon genetic modification techniques that led to the development of CRISPR technology and the obstacles present with delivery of the components necessary for gene editing. • Ormond, Kelly E et al. “Human Germline Genome Editing.” American journal of human genetics vol. 101,2 (2017): 167-176. doi:10.1016/j.ajhg.2017.06.012 A statement made by the American Society of Human Genetics about the use of genome-editing techniques like CRISPR-Cas9 for somatic and germline gene editing is explained in this review article. In summary, the statement was that germline gene editing should not be done if it ends in a pregnancy, germline gene editing in vitro on human embryos does not have to be prohibited, and that future applications of germline gene editing for medicine should not continue until there is ethical justification and a compelling reason. • Harrison, Patrick T, and Stephen Hart. “A beginner's guide to gene editing.” Experimental physiology vol. 103,4 (2018): 439-448. doi:10.1113/EP086047 This review discusses how gene editing has developed since the 1980s, leading to precise and relatively quick modification of genes using CRISPR-Cas9. It also summarizes important advancements with CRISPR resulting in an increase in efficiency and a reduction in off-target effects. • Banerjee, Budhaditya, and Richard I Sherwood. “A CRISPR view of gene regulation.” Current opinion in systems biology vol. 1 (2017): 1-8. doi:10.1016/j.coisb.2016.12.016 This article discusses how a reduction in costs is allowing genome sequencing of patients and different cancers more routine in medicine and how the effects of non-coding DNA on gene expression are not completely understood. It then summarizes how CRISPR-based gene editing is instrumental in predicting how DNA sequences affect gene regulation. • Robinson-Hamm, Jacqueline N, and Charles A Gersbach. “Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.” Human genetics vol. 135,9 (2016): 1029-40. doi:10.1007/s00439016-1725-z Advancements in gene therapy and editing, namely the CRISPR/Cas9 system, have shown promise in animal models and the cells of patients for restoration of the dystrophin protein expression for DMD treatment, but challenges remain. This review article describes the status of these techniques and considerations to make for their further development. • Uddin, Fathema et al. “CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future.” Frontiers in oncology vol. 10 1387. 7 Aug. 2020, doi:10.3389/fonc.2020.01387 This review article is about how CRISPR-Cas9 has revolutionized gene therapy for targeted gene editing by addressing some of the issues with traditional gene therapy, such as immunotoxicity and oncogenesis, it still has some limitations. Data from gene therapy trials is discussed, along with the best strategy for the effective and safe use of CRISPR-Cas9 in a clinical setting. • Min, Yi-Li et al. “CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells.” Science advances vol. 5,3 eaav4324. 6 Mar. 2019, doi:10.1126/sciadv.aav4324 This article is about an experiment in which CRISPR-Cas9 was used with an AAV9 vector to correct the deletion of exon 44 in the dystrophin gene in a mouse model and cardiomyocytes derived from patient induced pluripotent stem cells. The article also discusses the important dosages of the gene editing components for optimal gene correction in vivo for its clinical use in DMD treatment. • Long, Chengzu et al. “Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.” Science (New York, N.Y.) vol. 345,6201 (2014): 1184-1188. doi:10.1126/science.1254445 The experiment discussed in this article used CRISPR-Cas9 for the correction of a mutation in the dystrophin gene in the germline of a mouse model of DMD. Monitoring of muscle structure and function demonstrated 2 to 100% correction of the Dmd gene, exceeding gene correction efficiency. This strategy, along with certain technological advancements, shows promise for the correction of mutations causing DMD in muscle tissue of patients.
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Societal Implications of CRISPR-Cas9 as Treatment for Duchenne Muscular Dystrophy
Name
Institution
Professor
Course
Date

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Table of Contents
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Introduction ..................................................................................................................................... 3

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Pros/Cons of CRISPR-Cas9 as DMD Treatment ............................................................................ 4

3

2.1

Uses/Strengths/Benefits of the CRISPR/Cas9 ........................................................................ 5

2.2

Risks/Weaknesses/Drawbacks of the CRISPR/Cas9 .............................................................. 6

Ethics of the CRISPR-Cas9 Technology ........................................................................................ 8
3.1

Moral Aspect of CRISPR-Cas9 as DMD Treatment .............................................................. 8

3.1.1

Safety .............................................................................................................................. 9

3.1.2

Informed Consent.......................................................................................................... 10

3.2

Concerns ............................................................................................................................... 10

3.2.1
4

Financial Cost ............................................................................................................................... 11
4.1

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Justice and Equity ......................................................................................................... 10

Any Effects on Economy ...................................................................................................... 11

Conclusion .................................................................................................................................... 11

References ............................................................................................................................................. 12

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Introduction

Duchenne muscular dystrophy (DMD) refers to a severe form of muscular dystrophy
that is known to primarily affect boys (Robinson-Hamm et al., 2016). This disease is
characterized by muscle weaknesses that normally start around the age of four years.
However, the problem worsens quickly. Besides, with this disease, muscle loss normally
takes place in the thigh and the pelvis. The loss of muscles in these areas is followed by the
loss of muscles by the arms. DMD is a genetic disorder. Besides, the disease is characterized
by progressive and gradual weakness and degeneration of muscles as a result of changes and
alterations of a protein that is referred to as dystrophin (Robinson-Hamm et al., 2016). The
dystrophin protein is very important in people because it functions by keeping the muscle
cells intact. It should be noted that DMD is among the four conditions that are referred to as
dystrophinopathies. According to Min et al. (2019), DMD symptoms start in early childhood,
normally between the age of 2 and 3 years. Studies show that this disease majorly affects
boys. However, it also affects girls but in rare cases. As per Min et al. (2019), DMD is the
most common childhood-onset type of muscular dystrophy. Reports show that the birth
prevalence of the disease is estimated to be one in every 3,500 live male births (Min et al.
2019). One of the methods used for the treatment of this disease is CRISPR-Cas9. CRISPRCas9 is a unique treatment technology that enables medical researchers as well as geneticists
to edit parts of the genome (Uddin et al. 2020). They do this by primarily removing, adding
as well as altering sections or parts of th...


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