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Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.
Human genetic enhancement or human genetic engineering refers to human enhancement by means of a genetic modification. This could be done in order to cure diseases, prevent the possibility of getting a particular disease, to improve athlete performance in sporting events, or to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence. These genetic enhancements may or may not be done in such a way that the change is heritable.
A designer baby is a baby whose genetic makeup has been selected or altered, often to exclude a particular gene or to remove genes associated with disease. This process usually involves analysing a wide range of human embryos to identify genes associated with particular diseases and characteristics, and selecting embryos that have the desired genetic makeup; a process known as preimplantation genetic diagnosis. Screening for single genes is commonly practiced, and polygenic screening is offered by a few companies. Other methods by which a baby's genetic information can be altered involve directly editing the genome before birth, which is not routinely performed and only one instance of this is known to have occurred as of 2019, where Chinese twins Lulu and Nana were edited as embryos, causing widespread criticism.
CRISPR is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea. These sequences are derived from DNA fragments of bacteriophages that had previously infected the prokaryote. They are used to detect and destroy DNA from similar bacteriophages during subsequent infections. Hence these sequences play a key role in the antiviral defense system of prokaryotes and provide a form of acquired immunity. CRISPR is found in approximately 50% of sequenced bacterial genomes and nearly 90% of sequenced archaea.
The historical application of biotechnology throughout time is provided below in chronological order.
Shredding refers to the process in bioinformatics of taking assembled gene sequences and disassembling them into short sequences of usually 500 to 750 base pairs (bp). This is generally done for the purpose of taking the short shredded sequences and reapplying various analysis and bioinformatic techniques. Being able to cut DNA samples and then run them through gel electrophoresis to study each strand in order to help find cures for diseases or illnesses is also another purpose.
Jennifer Anne Doudna is an American biochemist who has done pioneering work in CRISPR gene editing, and made other fundamental contributions in biochemistry and genetics. Doudna was one of the first women to share a Nobel in the sciences. She received the 2020 Nobel Prize in Chemistry, with Emmanuelle Charpentier, "for the development of a method for genome editing." She is the Li Ka Shing Chancellor's Chair Professor in the department of chemistry and the department of molecular and cell biology at the University of California, Berkeley. She has been an investigator with the Howard Hughes Medical Institute since 1997.
Horizon Discovery Group plc ("Horizon"), is a gene editing company that designs and engineers genetically modified cells and then applies them in research and clinical applications in human health.
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Emmanuelle Marie Charpentier is a French professor and researcher in microbiology, genetics, and biochemistry. As of 2015, she has been a director at the Max Planck Institute for Infection Biology in Berlin. In 2018, she founded an independent research institute, the Max Planck Unit for the Science of Pathogens. In 2020, Charpentier and American biochemist Jennifer Doudna of the University of California, Berkeley, were awarded the Nobel Prize in Chemistry "for the development of a method for genome editing". This was the first science Nobel Prize ever won by two women only.
Editas Medicine, Inc.,, is a clinical-stage biotechnology company which is developing therapies for rare diseases based on CRISPR gene editing technology. Editas headquarters is located in Cambridge, Massachusetts and has facilities in Boulder, Colorado.
Intellia Therapeutics is a clinical-stage biotechnology company focused on developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. The company's in vivo programs use intravenously administered CRISPR as the therapy, in which the company's proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia's ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases.
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CRISPR gene editing is a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified. It is based on a simplified version of the bacterial CRISPR-Cas9 antiviral defense system. By delivering the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, allowing existing genes to be removed and/or new ones added in vivo.
Janice Chen is co-founder and chief technology officer of Mammoth Biosciences, a Brisbane, California-based company founded in 2018 that is developing diagnostic tests using CRISPR. She received her B.S. degree from Johns Hopkins University and as a graduate student at the University of California, Berkeley, she worked in the lab of CRISPR pioneer Jennifer Doudna, receiving her PhD in Molecular and Cell Biology.
Flagship Pioneering is an American life sciences venture capital company based in Cambridge, Massachusetts that invests in biotechnology, life sciences, health and sustainability companies. Portfolio companies include Moderna, Indigo Agriculture, Inari Agriculture and Novomer. The firm both funds and incubates companies.
Sherlock Biosciences is a biotechnology company based in Cambridge, Massachusetts developing diagnostic tests using CRISPR-Cas13. The company was founded in 2019 by Feng Zhang, Jim Collins, Omar Abudayyeh, and Jonathan Gootenberg of the Broad Institute.
Beam Therapeutics Inc. is an American biotechnology company conducting research in the field of gene therapies and genome editing. The company is headquartered in Cambridge, Massachusetts. In the development of therapies, the company relies on CRISPR and prime editing, whereby single nucleotides in a DNA sequence can be modified without cutting the DNA, theoretically reducing the likelihood of off-target effects compared to previous CRISPR-based methods.
Colossal Biosciences is a biotechnology and genetic engineering company working to de-extinct the woolly mammoth, the Tasmanian tiger, and the dodo. In 2023, it stated that it wants to have woolly mammoth hybrid calves by 2028, and wants to reintroduce them to the Arctic tundra habitat. Likewise, it plans to launch a thylacine research project to release Tasmanian tiger joeys back to their original Tasmanian and broader Australian habitat after a period of observation in captivity.
The Innovative Genomics Institute (IGI) is a nonprofit scientific research institute founded by Nobel laureate and CRISPR gene editing pioneer Jennifer Doudna and biophysicist Jonathan Weissman. The institute is based at the University of California, Berkeley, and also has member researchers at the University of California, San Francisco, UC Davis, UCLA, Lawrence Berkeley National Laboratory, Lawrence Livermore National Laboratory, Gladstone Institutes, and other collaborating research institutions. The IGI focuses on developing real-world applications of genome editing to address problems in human health, agriculture and climate change.
References
- ↑ Fannin, Rebecca (2022-03-12). "The 30-year-old female founder at the forefront of a billion-dollar bet on CRISPR gene editing". CNBC. Retrieved 2023-08-14.
- ↑ "News: Miniscule Cas nucleases do a Mammoth's job". CRISPR Medicine. Retrieved 2023-11-10.
- ↑ "Mammoth Biosciences". Craft.co. Archived from the original on 2021-05-27. Retrieved 2021-05-27.
- ↑ Isaacson, Walter (2021). The Code Breaker. Simon & Schuster. pp. 422–423. ISBN 978-1-9821-1585-2.
- ↑ "Horizon Discovery, Mammoth Biosciences Sign Second CRISPR Tools Development Agreement". Genomeweb. 2020-01-13. Retrieved 2021-05-27.
- ↑ Isaacson, Walter (2021). The Code Breaker. Simon & Schuster. pp. 427–433. ISBN 978-1-9821-1585-2.
- ↑ Mcardle, Matthew (May 17, 2021), Increasing SARS-CoV-2 testing capacity with CRISPR-based rapid testing, SelectScience, retrieved September 9, 2021
{{citation}}: CS1 maint: date and year (link) - ↑ "CRISPR unicorn Mammoth backs off diagnostics research, laying off 35 on testing team to focus on therapeutics". Endpoints News. Retrieved 2023-09-19.
- 1 2 Harrington, Lucas B.; Burstein, David; Chen, Janice S.; Paez-Espino, David; Ma, Enbo; Witte, Isaac P.; Cofsky, Joshua C.; Kyrpides, Nikos C.; Banfield, Jillian F.; Doudna, Jennifer A. (2018-11-16). "Programmed DNA destruction by miniature CRISPR-Cas14 enzymes". Science. 362 (6416): 839–842. Bibcode:2018Sci...362..839H. doi:10.1126/science.aav4294. ISSN 0036-8075. PMC 6659742 . PMID 30337455.
- 1 2 Pausch, Patrick; Al-Shayeb, Basem; Bisom-Rapp, Ezra; Tsuchida, Connor A.; Li, Zheng; Cress, Brady F.; Knott, Gavin J.; Jacobsen, Steven E.; Banfield, Jillian F.; Doudna, Jennifer A. (2020-07-17). "CRISPR-CasΦ from huge phages is a hypercompact genome editor". Science. 369 (6501): 333–337. Bibcode:2020Sci...369..333P. doi:10.1126/science.abb1400. ISSN 0036-8075. PMC 8207990 . PMID 32675376.
- ↑ Hart, Robert. "Mammoth Biosciences Inks $691 Million Deal With Vertex For Crispr Gene-Editing Therapies". Forbes. Retrieved 2023-09-19.
- ↑ Burger, Ludwig; Weiss, Patricia (2022-01-10). "Bayer in gene therapy collaboration with Mammoth Biosciences". Reuters. Retrieved 2023-09-19.
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