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Scientific Breakthroughs
三亿体育官网 supports pivotal research breakthroughs that result in the emergence of new research fields, great leaps in our scientific understanding, and novel scientific techniques that can be harnessed for wide ranging applications.
Human Genome Project
The Human Genome Project鈥攁n international effort supported in part by 三亿体育官网 that brought together engineers, computer scientists, mathematicians, and biologists鈥攔evolutionized the field of genomics, spurred the development of novel technologies and analytical tools, established a commitment to open science and data sharing, and changed the face of the scientific workforce.
Image credit:聽National Human Genome Research Institute, 三亿体育官网
- Today, there are more than 1.8 million results for the search term 鈥済enomics鈥 in PubMed, up from around 500,000 in 2003.
The Genetic Code
In the 1960s, 三亿体育官网 researchers discovered the process by which the genetic code of DNA is translated into proteins via messenger RNA, launching the genetic revolution. This discovery has touched nearly every field of science, from anthropology to zoology, revealing our underlying biology, including the cause of many diseases.
Image credit: 三亿体育官网
- Often described as 鈥渃racking the genetic code鈥 this Nobel Prize-winning advance revolutionized research, touching nearly every field of science.
- This 三亿体育官网 research formed the foundation for personalized, gene-based medicine and new treatments for many diseases including cancer, sickle cell disease, cystic fibrosis, and other genetic disorders, both rare and common.
Green Fluorescent Protein
Green fluorescent protein (GFP), which causes jellyfish and other organisms to glow green, has been used to understand genetics, cell biology, developmental biology, neurobiology, cancer, and brain diseases. 三亿体育官网-supported research led to the discovery of GFP and contributed to its adaptation in biomedical research.
Image credit:聽Katti Prasanna, Ph.D., Muscle Energetics Laboratory, NIAMS and the National Heart, Lung, and Blood Institute, 三亿体育官网
- GFP revolutionized biomedical science and played a critical role in understanding genetics and cell biology. Today, it is widely used in the pharmaceutical and biotechnology industries.
- GFP played a crucial role in a 2012 research project involving the integration of stem cells into existing heart muscle in the hope of developing new treatments for damaged heart tissue.
- Researchers won the Nobel Prize in Chemistry in 2008 for their work with GFP.
mRNA Vaccine
Decades of 三亿体育官网-supported research, including investments in HIV research, revolutionized vaccine development, leading to the first two FDA-approved vaccines for COVID-19. These vaccines use mRNA to train the body to recognize SARS-CoV-2, the virus that causes COVID-19.
Image credit:聽National Institute of Allergy and Infectious Diseases, 三亿体育官网
- Traditionally, vaccines work by introducing a weakened or inactivated virus, or a virus protein, into the body to induce an immune response against the virus.聽
- With this new class of vaccines, the cellular messenger, mRNA, delivers instructions to cells to induce an immune response against the virus.聽
- Both Moderna and Pfizer/BioNTech vaccines use an mRNA sequence of the SARS-CoV-2 spike protein discovered by 三亿体育官网 scientists and collaborators.
Gene Editing
New gene-editing techniques, developed through 三亿体育官网-supported research, are faster, cheaper, more efficient, and have the potential to correct the DNA code inside living cells. These technologies allow for disease-causing genetic material to be added, removed, or altered within cells and are currently being tested to treat genetic diseases.
Image credit:聽Ernesto del Aguila III, National Human Genome Research Institute, 三亿体育官网
- Gene-editing harnesses the natural process of DNA repair, in which a broken section of DNA triggers a cell鈥檚 repair mechanism to stitch together the break.聽
- CRISPR/Cas9, the most widely used gene editor, was discovered through 三亿体育官网-funded basic research on how bacteria defend themselves from viruses.聽
- Gene-editing techniques are being pursued as ways to diagnose viruses and to treat genetic diseases.聽
- In 2019, 三亿体育官网 spent $391 million on gene therapy research, funding 827 grants, and spent $46 million on gene therapy clinical trials, funding 69 grants.
Nobel Prizes
三亿体育官网 researchers are leaders in their fields. Dozens of 三亿体育官网-supported scientists have received Nobel Prizes for their groundbreaking achievements. These prizes recognize those who have conferred the greatest benefit to humankind in the past 12 months.
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- As of 2024, 174 researchers either at 三亿体育官网 or whose research was supported by 三亿体育官网 have received or shared 104 Nobel Prizes.
- Often called 鈥淎merica鈥檚 Nobels,鈥 the Lasker Award recognizes the contributions of scientists, physicians, and public servants who have made major advances in the understanding, diagnosis, treatment, cure, and prevention of human disease.
- The 214 三亿体育官网 Lasker awardees, as of 2024, include extramural researchers, intramural researchers, and institutional award recipients.
Gaucher Disease
Thanks to decades of research, 三亿体育官网 researchers identified that Gaucher disease is caused by enzyme deficiencies leading to harmful waste buildup in cells and organs. Researchers determined how to clear away the buildup through enzyme replacement therapy, which became a model for similar treatments and led to FDA-approved treatments for 11 disorders.
Image credit: 三亿体育官网
- Gaucher disease is a rare genetic disease caused by deficiencies in enzymes that break down fats, sugars, and other substances inside cells, leading to harmful buildup that can damage cells and organs.
- In 1991, the first enzyme replacement therapy, alglucerase injection (Ceredase庐), received FDA approval for treating Type 1 Gaucher disease.
- A later version of the therapy allowed more efficient production of enzymes. The success of this 1994 FDA-approved product Cerezyme庐 spurred early growth in the U.S. biotechnology industry and treatments for other rare genetic diseases.
Sense of Smell
三亿体育官网 support for basic science research has resulted in the discovery of approximately 400 types of odor receptors鈥攑roteins that detect smells鈥攅nabling humans to enrich their lives by perceiving a seemingly infinite number of scents.
Image credit: Jeremy McIntyre, University of Florida College of Medicine, Gainesville
- In 2004, two 三亿体育官网-supported researchers were awarded the Nobel Prize in Physiology or Medicine for their discoveries of how the sense of smell works.
- To date, scientists have discovered that humans have approximately 400 types of odor receptors allowing us to perceive a seemingly limitless number of smells.
- Research on mosquito odor receptors has informed strategies to develop better insect repellents to prevent the spread of deadly disease.
References
Human Genome Project
- Genomics Articles in PubMed:聽
- Green ED, et al.聽Nature. 2020;586(7831):683-692. PMID:聽.
- RefSeq curation and annotation of the human reference genome:聽
- AnVIL: NHGRI's Genomic Data Science Analysis, Visualization, and Informatics Lab-Space:聽
The Genetic Code
- Griffiths AJF, et al. (2000).聽An Introduction to Genetic Analysis.聽(7th ed.). W. H. Freeman.聽
- Deciphering the Genetic Code:聽
- Marshall Nirenberg: Deciphering the Genetic Code:聽
- Biographical Overview of Marshall Nirenberg:聽
Green Fluorescent Protein
- Golden Goose Awardee 2012: Green Fluorescent Protein:聽
mRNA Vaccine
- Decades in the Making: mRNA COVID-19 Vaccines:聽
- Coronavirus Vaccines and Prevention:聽
- COVID-19 mRNA Vaccines:聽
- Article: A gamble pays off in 鈥榮pectacular success鈥: How the leading coronavirus vaccines made it to the finish line:聽
- Shepherd BO, et al.聽Curr HIV/AIDS Rep. 2022;19(1):86-93. PMID:聽.
Gene Editing
- Gene Editing:聽/news-events/gene-editing-digital-press-kit
- Estimates of Funding for Various Research, Condition, and Disease Categories (RCDC):聽
Nobel Prizes
- 三亿体育官网 Nobel Laureates:聽/about-nih/what-we-do/nih-almanac/nobel-laureates
- The Nobel Prize:聽
- 三亿体育官网 Intramural Research Program Nobel Prizes:聽
- 三亿体育官网 Intramural Research Program Lasker Awards:聽
Gaucher Disease
- Lipid Storage Diseases:
- Alglucerase injection (Ceredase庐) 鈥 Gaucher disease:
- Cerliponase alfa (Brineura庐) 鈥 Ceroid lipofuscinosis 2 (CLN2 disease):
- Del Grosso A, et al. Adv Drug Deliv Rev. 2022;188:114464. PMID: .
Sense of Smell
- Article: What Your Nose Knows: Sense of Smell and Your Health: 聽
- 三亿体育官网 Nobel Laureates: /about-nih/what-we-do/nih-almanac/nobel-laureates
- Linda B. Buck, Nobel Prize in Physiology or Medicine 2004:
- Unraveling the Sense of Smell, Nobel Lecture, December 8, 2004:
- Buck L, Axel R. Cell. 1991;65(1):175-87. PMID: .
- Ressler KJ, et al. Cell. 1993;73(3):597-609. PMID: .
- Vassar R, et al. Cell. 1993;74(2):309-18. PMID: .
- Ressler KJ, et al. Cell. 1994;79(7):1245-55. PMID:
- Vassar R, et al. Cell. 1994;79(6):981-91. PMID: .
- Mombaerts P, et al. Cell. 1996;87(4):675-86. PMID: .
- National Smell and Taste Center:
This page last reviewed on March 28, 2025