In this article, we will explore how nanotechnology, including nanorobots and gene therapy, could revolutionize medicine by treating and preventing currently incurable diseases. |
Nanotechnology is a rapidly developing field with the potential to revolutionize many industries, including medicine. [1] Nanobots and gene therapy are two promising areas of nanotechnology that have the potential to revolutionize healthcare.
Nanobots are microscopic machines that can be programmed to perform specific tasks. They could be used to deliver drugs to targeted areas of the body, remove toxins, or repair damaged cells. Nanobots could also be used to diagnose diseases at an early stage, before they become serious.
Figure 01: Nanobots and gene therapy
Gene therapy is a treatment that involves inserting genes into cells to correct a genetic defect. This could be used to treat a variety of diseases, including cancer, cystic fibrosis, and sickle cell anemia.[1] Gene therapy is still in its early stages of development, but it has the potential to be a cure for many diseases that are currently incurable.
Figure 02: Gene Therapy
The combination of nanobots and gene therapy could have a profound impact on healthcare. Nanobots could deliver gene therapy treatments to targeted cells, making them more effective and less harmful.[3] Nanobots could also be used to monitor the progress of gene therapy treatments and make adjustments as needed.
In the future, nanobots and gene therapy could be used to treat a wide range of diseases, including:
Cancer
Heart disease
Stroke
Alzheimer's disease
Parkinson's disease
HIV/AIDS
Cystic fibrosis
Sickle cell anemia
These treatments could be more effective, less harmful, and more affordable than traditional treatments. They could also be used to prevent diseases from developing in the first place.
The development of nanobots and gene therapy is still in its early stages, but it is an exciting field with the potential to revolutionize healthcare. These technologies could one day make it possible to cure many diseases that are currently incurable.
What is the current state of gene therapy, and what are some of the most significant breakthroughs in this technology that brought us to this point?
One of the most significant breakthroughs in gene therapy was the development of viral vectors. Viral vectors are viruses that have been modified to carry therapeutic genes into cells. This allows scientists to deliver genes to cells in a targeted and efficient way.[5]
Another major breakthrough was the development of new gene editing technologies, such as CRISPR-Cas9. CRISPR-Cas9 is a tool that can be used to precisely edit DNA sequences. This has made it possible to correct genetic defects that cause diseases.
In 2017, the FDA approved the first gene therapy treatment for a genetic disease, Luxturna. Luxturna is used to treat a rare form of blindness called Leber congenital amaurosis. This approval was a major milestone for gene therapy and showed that it can be used to treat real diseases in people. [7]
Since then, there have been a number of other gene therapy treatments approved for use in humans. These include Zolgensma, which is used to treat spinal muscular atrophy, and Kymriah, which is used to treat leukemia.
Gene therapy is still in its early stages of development, but it has the potential to revolutionize the treatment of many diseases. [9] With continued research and development, we can expect to see even more gene therapy treatments approved in the years to come.
Here are some of the most significant breakthroughs in gene therapy that brought us to this point:
The discovery of the double helix structure of DNA in 1953. This discovery paved the way for our understanding of how genes work and how they can be manipulated.
The first successful gene therapy experiments in animals in the 1970s. These experiments showed that it was possible to insert genes into cells and have them function properly. [11]
The development of viral vectors in the 1980s. Viral vectors made it possible to deliver genes to cells in a more efficient and targeted way.
The completion of the human genome project in 2003. This gave scientists a complete map of the human genome, which is essential for gene therapy research.
The development of new gene editing technologies, such as CRISPR-Cas9, in the 2010s. CRISPR-Cas9 is a powerful tool that can be used to precisely edit DNA sequences, making it possible to correct genetic defects that cause diseases [24].
These are just some of the most significant breakthroughs in gene therapy that have brought us to this point. With continued research and development, we can expect to see even more breakthroughs in the years to come [22].
What is the process for reading genes, turning them on/off, and what are the effects?
The process of reading genes, turning them on/off, and what are the effects is called gene regulation. It is a complex process that involves a variety of proteins and molecules [20].
Figure 03: How cell reads Genes.
The first step in gene regulation is transcription. In transcription, the DNA sequence of a gene is copied into a messenger RNA (mRNA) molecule [18]. The mRNA molecule then leaves the nucleus of the cell and travels to the cytoplasm, where it is translated into a protein. [13]
The process of turning genes on and off is called regulation of transcription. There are a number of different ways that genes can be regulated, including:
DNA methylation: This is the addition of a methyl group to a DNA molecule. Methyl groups can silence genes by preventing them from being transcribed.
Histone modification: This is the chemical modification of histone proteins, which are associated with DNA. Histone modifications can affect how tightly DNA is packed, which can affect gene expression [16].
RNA interference (RNAi): This is a process by which small RNA molecules can silence genes by binding to mRNA molecules and preventing them from being translated into proteins.
The effects of gene regulation can be widespread. For example, if a gene is turned off, it will not be translated into a protein, which can have a number of different effects on the cell [15]. In some cases, turning off a gene can be beneficial, such as when it is used to treat a disease. In other cases, turning off a gene can be harmful, such as when it leads to cancer [14].
Benefits of gene therapy:
Gene therapy can potentially cure diseases that are currently incurable, such as cystic fibrosis, sickle cell anemia, and cancer [12].
Gene therapy can be used to treat diseases that are not well-controlled by other treatments, such as HIV/AIDS and hemophilia [17].
Gene therapy can be used to prevent diseases from developing in the first place [10].
Gene therapy is a relatively new technology, so there is still room for improvement. As research continues, we can expect to see even more benefits from gene therapy in the future.
Risks of gene therapy:
Gene therapy can cause side effects, such as inflammation, infection, and cancer [8].
Gene therapy can be unpredictable. It is not always clear how genes will be expressed after they have been inserted into cells [19].
Gene therapy can be expensive. The cost of developing and manufacturing gene therapy treatments is high [6].
Gene therapy is still in its early stages of development. There is not enough data to know how safe and effective it is in the long term.
Adverse events:
There have been a number of adverse events reported in gene therapy trials, including:
Inflammation: This is the most common side effect of gene therapy. It can occur at the injection site or throughout the body [4].
Infection: Gene therapy can introduce viruses or other pathogens into the body. This can lead to infection.
Cancer: Gene therapy can increase the risk of cancer. This is because it can introduce genes that are associated with cancer into cells [2].
Death: There have been a few cases of death in gene therapy trials. This is usually due to complications from other side effects, such as inflammation or infection [21].
It is important to note that the risks of gene therapy are outweighed by the potential benefits for some diseases. However, it is important to weigh the risks and benefits carefully before deciding whether or not to undergo gene therapy treatment [29].
What is the future outlook in the decades ahead?
The future outlook for gene therapy is very promising. With continued research and development, we can expect to see gene therapy treatments approved for a wider range of diseases in the coming decades. Applications of gene therapy in the future:
Cure for genetic diseases: Gene therapy has the potential to cure a wide range of genetic diseases, such as cystic fibrosis, sickle cell anemia, and cancer.
Treatment for acquired diseases: Gene therapy can also be used to treat acquired diseases, such as HIV/AIDS, hemophilia, and Alzheimer's disease [30].
Prevention of diseases: Gene therapy can be used to prevent diseases from developing in the first place. For example, gene therapy could be used to vaccinate people against diseases, such as malaria and HIV/AIDS [28].
Enhancement of human capabilities: Gene therapy could be used to enhance human capabilities, such as intelligence, strength, and endurance. However, this is a controversial topic, and there are ethical concerns about using gene therapy for enhancement purposes [27].
Figure 04: Gene Therapy Market Size, Share - Global Industry Growth Report 2028
Overall, the future outlook for gene therapy is very promising. With continued research and development, we can expect to see gene therapy treatments becoming more common and affordable in the coming decades. This could revolutionize the way we treat diseases and improve the quality of life for millions of people around the world [25].
How gene therapy could be used in the future:
Cystic fibrosis: Gene therapy is currently being used to treat cystic fibrosis. This is a genetic disease that affects the lungs and digestive system. Gene therapy can help to improve lung function and reduce the symptoms of cystic fibrosis.
Sickle cell anemia: Gene therapy is also being used to treat sickle cell anemia. This is a genetic disease that affects the blood cells. Gene therapy can help to reduce the number of sickle cells and improve the quality of life for people with sickle cell anemia [23].
Cancer: Gene therapy is being investigated as a treatment for cancer. This is because gene therapy can target cancer cells and kill them without harming healthy cells.
HIV/AIDS: Gene therapy is being investigated as a treatment for HIV/AIDS. This is because gene therapy can help to prevent the virus from replicating [23].
Hemophilia: Gene therapy is being investigated as a treatment for hemophilia. This is a genetic disease that affects the blood clotting system. Gene therapy can help to provide people with hemophilia with the ability to clot their blood normally.
These are just a few examples of how gene therapy could be used in the future. With continued research and development, we can expect to see even more applications for gene therapy in the years to come.
About the Author:
Maheen Javed, M.D. graduated as a medical doctor in 2020 with experience in medical research, medical writing and other diverse areas in the medical field. She currently practices in a hospital and works as a professional medical writer and researcher, writing technical articles on a wide variety of topics in the medical field, such as mental health, diabetes, women's health, cancer research, psychiatry, neurology, surgery and mental health.
About the editor:
Brian Hoy has over 20 years of experience in the medical device industry and business formation, supporting the full lifecycle with global scope. Brian consults for industry and provides general advisory and off-hours support.
References:
1. Mukherjee S. Mukherjee S. The gene: an intimate history. Nova York: Scribner; 2016. Genetic therapies: posthuman gene therapy.415 Chap. 34.
2. Friedmann T. A brief history of gene therapy. Nat Genet. 1992;2(2):93–98. Review.
3. Misra S. Human gene therapy: a brief overview of the genetic revolution. J Assoc Physicians India. 2013;61(2):127–133. Review.
4. Tebas P, Stein D, Tang WW, Frank I, Wang SQ, Lee G, et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med. 2014;370(10):901–910.
5. Linden R. Gene therapy: what it is, what it is not and what it will be. Estud Av. 2010;24(70):31–69.
6. Ginter EK. Gene therapy of hereditary diseases. Vopr Med Khim. 2000;46(3):265–278. Review. Russian.
7. Mathews QL, Curiel DT. Gene therapy: human germline genetics modifications-assessing the scientific, socioethical, and religious issues. South Med J. 2007;100(1):98–100.
8. Bank A. Human somatic cell gene therapy. Bioessays. 1996;18(12):999–1007. Review.
9. Gardlík R, Pálffy R, Hodosy J, Lukács J, Turna J, Celec P. Vectors and delivery systems in gene therapy. Med Sci Monit. 2005;11(4):RA110–RA121. Review.
10. McDonnell WM, Askari FK. DNA vaccines. N Engl J Med. 1996;334(1):42–45. Review.
11. Plank C, Tang MX, Wolfe AR, Szoka FC., Jr Branched cationic peptides for gene delivery: role of type and number of cationic residues in formation and in vitro activity of DNA polyplexes. Hum Gene Ther. 1999;10(2):319–332. Erratum in: Hum Gene Ther. 1999;10(13):2272.
12. Caplen NJ, Kinrade E, Sorgi F, Gao X, Gruenert D, Geddes D, et al. In vitro liposome-mediated DNA transfection of epithelial cell lines using the cationic liposome DC-Chol/DOPE. Gene Ther. 1995;2(9):603–613.
13. Nabel GJ, Chang AE, Nabel EG, Plautz GE, Ensminger W, Fox BA, et al. Immunotherapy for cancer by direct gene transfer into tumors. Hum Gene Ther. 1994;5(1):57–77.
14. Yang Y, Nunes FA, Berencsi K, Furth EE, Gönczöl E, Wilson JM. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci U S A. 1994;91(10):4407–4411.
15. Kay MA. State-of-the-art gene-based therapies: the road ahead. Nat Rev Genet. 2011;12(5):316–328. Review.
16. Eshhar Z, Waks T, Gross G, Schindler DG. Specific activation and targeting of cytotoxic lymphocytes through chimeric single chains consisting of antibody-binding domains and the gamma or zeta subunits of the immunoglobulin and T-cell receptors. Proc Natl Acad Sci U S A. 1993;90(2):720–724.
17. Gross G, Waks T, Eshhar Z. Expression of immunoglobulin-T-cell receptor chimeric molecules as functional receptors with antibody-type specificity. Proc Natl Acad Sci U S A. 1989;86(24):10024–10028.
18. Parham P. Parham P. The immune system. 3rd. Nova York: Garland Science; 2009. Antigen recognition by T lymphocytes.113 Chap. 5.
19. Chambers CA, Allison JP. Co-stimulation in T cell responses. Curr Opin Immunol. 1997;9(3):396–404. Review.
20. Wilkins O, Keeler AM, Flotte TR. CAR T-Cell Therapy: Progress and Prospects. Hum Gene Ther Methods. 2017;28(2):61–66. Review.
21. Zhong XS, Matsushita M, Plotkin J, Riviere I, Sadelain M. Chimeric antigen receptors combining 4-1BB and CD28 signaling domains augment PI3kinase/AKT/Bcl-XL activation and CD8+ T cell-mediated tumor eradication. Mol Ther. 2010;18(2):413–420.
22. Maude SL, Frey N, Shaw PA, Aplenc R, Barrett DM, Bunin NJ, et al. Chimeric antigen receptor T cells for sustained remissions in leukemia. N Engl J Med. 2014;371(16):1507–1517.
23. Lee DW, Gardner R, Porter DL, Louis CU, Ahmed N, Jensen M, et al. Current concepts in the diagnosis and management of cytokine release syndrome. Blood. 2014;124(2):188–195. Erratum in: Blood. 2015;126(8):1048. Dosage error in article text.
24. Marraffini LA, Sontheimer EJ. CRISPR interference: RNA-directed adaptive immunity in bacteria and archaea. Nat Rev Genet. 2010;11(3):181–190. Review.
25. Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012;337(6096):816–821.
26. Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339(6121):819–823.
27. Vieira GV, Cecílio NT, Arruda LM, Sales KU. Visão geral do mecanismo básico de ação. In: Introdução à técnica de CRISPR. Ribeirão Preto: Cubo; 2016. 54 Cap.
28. Gori JL, Hsu PD, Maeder ML, Shen S, Welstead GG, Bumcrot D. Delivery and Specificity of CRISPR/Cas9 Genome Editing Technologies for Human Gene Therapy. Hum Gene Ther. 2015;26(7):443–451. Review.
29. De Witt MA, Magis W, Bray NL, Wang T, Berman JR, Urbinati F, et al. Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells. Sci Transl Med. 2016;8(360) 360ra134.
30. Callaway E. Second Chinese team reports gene editing in human embryos. Nature. 2016 Apr 08; doi: 10.1038/nature.2016.19718. 2016.
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