Скачать или смотреть Should we use Gene Therapy to cure genetic disorders?

Gene editing is being tested for the treatment of genetic diseases such as cystic fibrosis, sickle cell anemia, blood disorders, and cancers. Genetic engineering techniques such as CRISPR allow targeted correcting of DNA mutations.

Should genetic engineering be used to correct DNA mutations causing disease?

Mutations, or changes in the information coded within your DNA, can cause the production of incorrect proteins contributing to a variety of genetic diseases. Within the cell, the DNA stays secured and protected within the nucleus while temporary RNA copies are used to direct the cell in the building of proteins. Using three-letter words to visualize how changes to the DNA information will result in abnormal proteins that may not function properly. Clinical trials are underway to use a temporary RNA copy to direct the affected cells to produce the proper, working proteins and ideally treat the cause of the disease. RNA treatments are temporary, requiring regular treatments. Recent development of CRISPR, a powerful molecular scissors allowing targeted changes to the DNA of a cell, can provide a tool to permanently cure genetic diseases. In the current news are reports of individuals cured of sickle cell anemia using CRISPR technology to alter the DNA of blood stem cells in the laboratory before transplanting back into the patient. However, potential use of genetic engineering in this way remains controversial.

Ethical Dilemma in Science
Should genetic engineering be used to correct DNA mutations causing disease?
Should genetic engineering be used to treat all genetic disorders or only those that are lethal?
How do we ensure equitable access to the benefits of genetic engineering?
What would you need to know to make a decision about the use of genetic engineering for yourself or a loved one?

0:00 Introduction
1:09 Current Events
2:21 DNA, RNA, and proteins
4:11 Explaining DNA, RNA, and proteins
5:19 Gene mutation
6:00 Cystic Fibrosis
7:26 RNA treatment
8:11 RNA treatment for Cystic Fibrosis
9:24 Genetic engineering using CRISPR
10:38 Nobel Prize in Chemistry 2020
11:18 Woman "cured" of sickle cell anemia using genetic engineering
13:36 Ethical Dilemma in Science

Recommended Readings:
Brown, G. (2022) CRISPR's quest to slay Donegal Amy. Wired. https://www.wired.com/story/crispr-tr...

Mullin, E. (2022) The era of one-shot, multimillion-dollar genetic cures is here. Wired. https://www.wired.com/story/the-era-o...

Additional References:

Anonymous (2023). Cystic Fibrosis Foundation funds nanoparticle delivery to lung. Genetic Engineering & Biotechnology News. https://www.genengnews.com/topics/omi...

Grillo, J. (2022) Healing breath: researchers dramatically improve inhalable mRNA therapy. Phys.org. https://phys.org/news/2022-12-inhalab...

Hubrecht Institute (2021) New CRISPR/Cas9 technique corrects cystic fibrosis in human cultured stem cells. Science Daily. https://www.sciencedaily.com/releases...

Kupferschmidt, K. (2023) Gene-editing summit touts sickle cell success, while questions on embryo editing linger. Science 379(6637) https://www.science.org/content/artic...

National Heart, Lung, and Blood Institute (2022) What is cystic fibrosis? https://www.nhlbi.nih.gov/health/cyst...

Royal Swedish Academy of Sciences (2020) Nobel Prize in Chemistry 2020, Popular information: Genetic scissors: a tool for rewriting the code of life. https://www.nobelprize.org/prizes/che...

Saey, T. H. (2020) Gene-editing tool CRISPR wins the chemistry Nobel. Science News. https://www.sciencenews.org/article/n...

Synthego. (2022) Sickle cell gene therapy using CRISPR. https://www.synthego.com/crispr-sickl...

Third International Summit on Human Genome Editing (2023) Statement from the Organising Committee of the Third International Summit on Human Genome Editing. The Royal Society. https://royalsociety.org/news/2023/03...

Thrasher, A. et al., (2016) On Human Gene Editing: International Summit Statement by the Organizing Committee. Issues in Science and Technology 32(3): https://issues.org/on-human-gene-edit...

Vaidyanathan, S., & Ryan, A. L. (2022). Editorial: Genome Editing to Treat Cystic Fibrosis and Other Pulmonary Diseases. Frontiers in genome editing, 4, 917916. https://www.frontiersin.org/articles/...

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