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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.
Herpes simplex research includes all medical research that attempts to prevent, treat, or cure herpes, as well as fundamental research about the nature of herpes. Examples of particular herpes research include drug development, vaccines and genome editing. HSV-1 and HSV-2 are commonly thought of as oral and genital herpes respectively, but other members in the herpes family include chickenpox (varicella/zoster), cytomegalovirus, and Epstein-Barr virus. There are many more virus members that infect animals other than humans, some of which cause disease in companion animals or have economic impacts in the agriculture industry.
Jennifer Anne Doudna is an American biochemist who has pioneered work in CRISPR gene editing, and made other fundamental contributions in biochemistry and genetics. 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.
HIV/AIDS research includes all medical research that attempts to prevent, treat, or cure HIV/AIDS, as well as fundamental research about the nature of HIV as an infectious agent and AIDS as the disease caused by HIV.
Geoffrey von Maltzahn is an American biological engineer and businessman in the biotechnology and life sciences industry. Von Maltzahn has cofounded companies including Indigo Agriculture, Generate Biomedicines, Seres Therapeutics, Tessera Therapeutics, and Quotient Therapeutics.
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.
Human germline engineering is the process by which the genome of an individual is edited in such a way that the change is heritable. This is achieved by altering the genes of the germ cells, which then mature into genetically modified eggs and sperm. For safety, ethical, and social reasons, there is broad agreement among the scientific community and the public that germline editing for reproduction is a red line that should not be crossed at this point in time. There are differing public sentiments, however, on whether it may be performed in the future depending on whether the intent would be therapeutic or non-therapeutic.
Intellia Therapeutics, Inc. is an American 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.
Aaron James Traywick was an American businessman and life extension activist in the transhumanism and biohacking communities. He sought to develop gene therapies to make inexpensive treatments available for incurable conditions such as AIDS and the herpes simplex virus. His lack of any medical training and his unconventional methods—such as broadcasting an associate injecting himself with an "untested experimental gene therapy", then later doing the same to himself in an onstage public demonstration—drew widespread criticism.
The He Jiankui affair is a scientific and bioethical controversy concerning the use of genome editing following its first use on humans by Chinese scientist He Jiankui, who edited the genomes of human embryos in 2018. He became widely known on 26 November 2018 after he announced that he had created the first human genetically edited babies. He was listed in Time magazine's 100 most influential people of 2019. The affair led to ethical and legal controversies, resulting in the indictment of He and two of his collaborators, Zhang Renli and Qin Jinzhou. He eventually received widespread international condemnation.
He Jiankui is a Chinese biophysicist. He was named as the inaugural director of the Institute of Genetic Medicine at Wuchang Technical College, a private undergraduate college in Wuhan, in September 2023. Before January 2019, He served as associate professor at the Department of Biology of the Southern University of Science and Technology (SUSTech) in Shenzhen, Guangdong, China. Earning a PhD from Rice University in Texas on protein evolution, including that of CRISPR, He learned gene-editing techniques (CRISPR/Cas9) as a postdoctoral researcher at Stanford University in California.
CRISPR gene editing (CRISPR, pronounced "crisper", refers to "clustered regularly interspaced short palindromic repeats") 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.
Cure Rare Disease is a non-profit biotechnology company based in Boston, Massachusetts that is working to create novel therapeutics using gene therapy, gene editing and antisense oligonucleotides to treat people impacted by rare and ultra-rare genetic neuromuscular conditions.
CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases. The company has approximately 500 employees and has offices in Zug, Switzerland, Boston, Massachusetts, San Francisco, California and London, United Kingdom. Its manufacturing facility in Framingham, Massachusetts won the Facilities of the Year Award (FOYA) award in 2022. The company’s lead program, exagamglogene autotemcel, or exa-cel, was granted regulatory approval by the US Food and Drug Administration (FDA) in December 2023.
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.
Howard E. Gendelman is an American physician-scientist whose research intersects the disciplines of neuroimmunology, pharmacology, and infectious diseases. Gendelman was born in Philadelphia, Pennsylvania. His research is focused on harnessing immune responses for therapeutic gain in HIV/AIDS and Neurodegenerative disease. He is the Margaret R. Larson Professor of infectious diseases and internal medicine at the University of Nebraska Medical Center (UNMC) in Omaha.
Precision BioSciences, Inc. is a publicly traded American clinical stage gene editing company headquartered in Durham, North Carolina. Founded in 2006, Precision is focused on developing both in vivo and ex vivo gene editing therapies using its proprietary "ARCUS" genome editing platform.
The Innovative Genomics Institute (IGI) is an American 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
- ↑ Sheldon, Cormac (30 October 2023). "Armed with gene technologies and CAR-Ts, scientists are attempting to eliminate viruses that escape immune detection and lurk in tissues for years". Nature Medicine . 41 (11): 1491–1493. doi:10.1038/s41587-023-02022-0. S2CID 264673741 . Retrieved 16 November 2023.
- ↑ "The first human study has launched to treat HIV patients with CRISPR. The CEO of the gene-editing startup says it could be a one-time cure". Business Insider . 27 January 2023. Retrieved 16 November 2023.
- ↑ Brzyski, Laura (5 March 2023). "Excision BioTherapeutics Has Secured $60M to Proceed with HIV Clinical Trial". Philadelphia . Retrieved 16 November 2023.
- ↑ Krieger, Lisa (28 October 2023). "CRISPR gene editing could kill HIV. But is it a cure?". The Mercury News . Retrieved 16 November 2023.
- ↑ Leuty, Ron (21 July 2023). "S.F. biotech inches closer to potential HIV cure with key move by FDA". San Francisco Business Times . Retrieved 17 November 2023.
- ↑ "Three people were gene-edited in an effort to cure their HIV. The result is unknown". MIT Technology Review. Retrieved 2024-03-20.
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