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<p class="pretitle"><a href="/about-nih/what-we-do/impact-nih-research/revolutionizing-science">Revolutionizing Science</a></p><div class='syndicate'><h1>Scientific Breakthroughs</h1><div><p>NIH 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.</p>
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<h3>Human Genome Project</h3>
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<p>The Human Genome Project—an international effort supported in part by NIH that brought together engineers, computer scientists, mathematicians, and biologists—revolutionized 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.</p>
<p class="credit">Image credit: National Human Genome Research Institute, NIH</p>
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<li>Today, there are more than 1.8 million results for the search term “genomics” in PubMed, up from around 500,000 in 2003.</li>
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<h3>The Genetic Code</h3>
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<p>In the 1960s, NIH 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.</p>
<p class="credit">Image credit: NIH</p>
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<li>Often described as “cracking the genetic code” this Nobel Prize-winning advance revolutionized research, touching nearly every field of science.</li>
<li>This NIH 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.</li>
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<h3>Green Fluorescent Protein</h3>
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<p>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. NIH-supported research led to the discovery of GFP and contributed to its adaptation in biomedical research.</p>
<p class="credit">Image credit: Katti Prasanna, Ph.D., Muscle Energetics Laboratory, NIAMS and the National Heart, Lung, and Blood Institute, NIH</p>
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<li>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.</li>
<li>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.</li>
<li>Researchers won the Nobel Prize in Chemistry in 2008 for their work with GFP.</li>
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<h3>mRNA Vaccine</h3>
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<p>Decades of NIH-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.</p>
<p class="credit">Image credit: National Institute of Allergy and Infectious Diseases, NIH</p>
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<li>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. </li>
<li>With this new class of vaccines, the cellular messenger, mRNA, delivers instructions to cells to induce an immune response against the virus. </li>
<li>Both Moderna and Pfizer/BioNTech vaccines use an mRNA sequence of the SARS-CoV-2 spike protein discovered by NIH scientists and collaborators.</li>
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<h3>Gene Editing</h3>
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<p>New gene-editing techniques, developed through NIH-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.</p>
<p class="credit">Image credit: Ernesto del Aguila III, National Human Genome Research Institute, NIH</p>
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<li>Gene-editing harnesses the natural process of DNA repair, in which a broken section of DNA triggers a cells repair mechanism to stitch together the break. </li>
<li>CRISPR/Cas9, the most widely used gene editor, was discovered through NIH-funded basic research on how bacteria defend themselves from viruses. </li>
<li>Gene-editing techniques are being pursued as ways to diagnose viruses and to treat genetic diseases. </li>
<li>In 2019, NIH spent $391 million on gene therapy research, funding 827 grants, and spent $46 million on gene therapy clinical trials, funding 69 grants.</li>
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<h3>Nobel Prizes</h3>
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<p>NIH researchers are leaders in their fields. Dozens of NIH-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.</p>
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<li>As of 2024, 174 researchers either at NIH or whose research was supported by NIH have received or shared 104 Nobel Prizes.</li>
<li>Often called “Americas Nobels,” 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.</li>
<li>The 214 NIH Lasker awardees, as of 2024, include extramural researchers, intramural researchers, and institutional award recipients.</li>
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<h3>Gaucher Disease</h3>
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<p>Thanks to decades of research, NIH 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.</p>
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<li>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.</li>
<li>In 1991, the first enzyme replacement therapy, alglucerase injection (Ceredase®), received FDA approval for treating Type 1 Gaucher disease.</li>
<li>A later version of the therapy allowed more efficient production of enzymes. The success of this 1994 FDA-approved product Cerezyme® spurred early growth in the U.S. biotechnology industry and treatments for other rare genetic diseases.</li>
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<h3>Sense of Smell</h3>
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<p>NIH support for basic science research has resulted in the discovery of approximately 400 types of odor receptors—proteins that detect smells—enabling humans to enrich their lives by perceiving a seemingly infinite number of scents.</p>
<p class="credit">Image credit: Jeremy McIntyre, University of Florida College of Medicine, Gainesville</p>
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<li>In 2004, two NIH-supported researchers were awarded the Nobel Prize in Physiology or Medicine for their discoveries of how the sense of smell works.</li>
<li>There are more smells than odor receptors, and its estimated that the number of odors that people can detect is somewhere between 10,000 and 100 billion, or even more.</li>
<li>Research on mosquito odor receptors has informed strategies to develop better insect repellents to prevent the spread of deadly disease.</li>
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<h2>References</h2>
<h3>Human Genome Project</h3>
<ol>
<li>Genomics Articles in PubMed: <a href="https://pubmed.ncbi.nlm.nih.gov/?term=genomics">https://pubmed.ncbi.nlm.nih.gov/?term=genomics</a></li>
<li>Green ED, et al. <em>Nature</em>. 2020;586(7831):683-692. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/33116284/">33116284</a>.</li>
<li>RefSeq curation and annotation of the human reference genome: <a href="https://www.ncbi.nlm.nih.gov/refseq/about/human/">https://www.ncbi.nlm.nih.gov/refseq/about/human/</a></li>
<li>AnVIL: NHGRI's Genomic Data Science Analysis, Visualization, and Informatics Lab-Space: <a href="https://anvilproject.org/">https://anvilproject.org/</a></li>
</ol>
<h3>The Genetic Code</h3>
<ol>
<li>Griffiths AJF, et al. (2000). <em>An Introduction to Genetic Analysis.</em> (7th ed.). W. H. Freeman. </li>
<li>Deciphering the Genetic Code: <a href="https://www.acs.org/content/acs/en/education/whatischemistry/landmarks/geneticcode.html">https://www.acs.org/content/acs/en/education/whatischemistry/landmarks/geneticcode.html</a></li>
<li>Marshall Nirenberg: Deciphering the Genetic Code: <a href="https://history.nih.gov/display/history/Nirenberg+Introduction">https://history.nih.gov/display/history/Nirenberg+Introduction</a></li>
<li>Biographical Overview of Marshall Nirenberg: <a href="https://profiles.nlm.nih.gov/spotlight/jj/feature/biographical">https://profiles.nlm.nih.gov/spotlight/jj/feature/biographical</a></li>
</ol>
<h3>Green Fluorescent Protein</h3>
<ol>
<li>Golden Goose Awardee 2012: Green Fluorescent Protein: <a href="https://www.goldengooseaward.org/01awardees/gfp">https://www.goldengooseaward.org/01awardees/gfp</a></li>
</ol>
<h3>mRNA Vaccine</h3>
<ol>
<li>Decades in the Making: mRNA COVID-19 Vaccines: <a href="https://covid19.nih.gov/nih-strategic-response-covid-19/decades-making-mrna-covid-19-vaccines">https://covid19.nih.gov/nih-strategic-response-covid-19/decades-making-mrna-covid-19-vaccines</a></li>
<li>Coronavirus Vaccines and Prevention: <a href="https://www.niaid.nih.gov/diseases-conditions/coronavirus-vaccines-prevention">https://www.niaid.nih.gov/diseases-conditions/coronavirus-vaccines-prevention</a></li>
<li>COVID-19 mRNA Vaccines: <a href="https://www.niaid.nih.gov/sites/default/files/mRNA%20Vaccine%20Development.pdf">https://www.niaid.nih.gov/sites/default/files/mRNA%20Vaccine%20Development.pdf</a></li>
<li>Article: A gamble pays off in spectacular success: How the leading coronavirus vaccines made it to the finish line: <a href="https://www.washingtonpost.com/health/2020/12/06/covid-vaccine-messenger-rna/">https://www.washingtonpost.com/health/2020/12/06/covid-vaccine-messenger-rna/</a></li>
<li>Shepherd BO, et al. <em>Curr HIV/AIDS Rep</em>. 2022;19(1):86-93. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/35089535/">35089535</a>.</li>
</ol>
<h3>Gene Editing</h3>
<ol>
<li>Gene Editing: <a href="https://www.nih.gov/news-events/gene-editing-digital-press-kit">https://www.nih.gov/news-events/gene-editing-digital-press-kit</a></li>
<li>Estimates of Funding for Various Research, Condition, and Disease Categories (RCDC): <a href="https://report.nih.gov/funding/categorical-spending">https://report.nih.gov/funding/categorical-spending#/</a></li>
</ol>
<h3>Nobel Prizes</h3>
<ol>
<li>NIH Nobel Laureates: <a href="https://www.nih.gov/about-nih/what-we-do/nih-almanac/nobel-laureates">https://www.nih.gov/about-nih/what-we-do/nih-almanac/nobel-laureates</a></li>
<li>The Nobel Prize: <a href="https://www.nobelprize.org/">https://www.nobelprize.org/</a></li>
<li>NIH Intramural Research Program Nobel Prizes: <a href="https://irp.nih.gov/about-us/honors/nobel-prize">https://irp.nih.gov/about-us/honors/nobel-prize</a></li>
<li>NIH Intramural Research Program Lasker Awards: <a href="https://irp.nih.gov/about-us/honors/lasker-award">https://irp.nih.gov/about-us/honors/lasker-award</a></li>
</ol>
<h3>Gaucher Disease</h3>
<ol>
<li>Lipid Storage Diseases: <a href="https://www.ninds.nih.gov/health-information/disorders/lipid-storage-diseases">https://www.ninds.nih.gov/health-information/disorders/lipid-storage-diseases</a></li>
<li>Alglucerase injection (Ceredase®) Gaucher disease: <a href="https://www.ninds.nih.gov/about-ninds/what-we-do/impact/ninds-contributions-approved-therapies/alglucerase-injection-ceredaser-gaucher-disease">https://www.ninds.nih.gov/about-ninds/what-we-do/impact/ninds-contributions-approved-therapies/alglucerase-injection-ceredaser-gaucher-disease</a></li>
<li>Cerliponase alfa (Brineura®) Ceroid lipofuscinosis 2 (CLN2 disease): <a href="https://www.ninds.nih.gov/about-ninds/what-we-do/impact/ninds-contributions-approved-therapies/cerliponase-alfa-brineurar-ceroid-lipofuscinosis-2-cln2-disease">https://www.ninds.nih.gov/about-ninds/what-we-do/impact/ninds-contributions-approved-therapies/cerliponase-alfa-brineurar-ceroid-lipofuscinosis-2-cln2-disease</a></li>
<li>Del Grosso A, et al. <em>Adv Drug Deliv Rev.</em> 2022;188:114464. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/35878795/">35878795</a>.</li>
</ol>
<h3>Sense of Smell</h3>
<ol>
<li>Article: What Your Nose Knows: Sense of Smell and Your Health:  <a href="https://newsinhealth.nih.gov/2016/08/what-your-nose-knows">https://newsinhealth.nih.gov/2016/08/what-your-nose-knows</a></li>
<li>NIH Nobel Laureates: <a href="https://www.nih.gov/about-nih/what-we-do/nih-almanac/nobel-laureates">https://www.nih.gov/about-nih/what-we-do/nih-almanac/nobel-laureates</a></li>
<li>Linda B. Buck, Nobel Prize in Physiology or Medicine 2004: <a href="https://www.nobelprize.org/womenwhochangedscience/stories/linda-buck">https://www.nobelprize.org/womenwhochangedscience/stories/linda-buck</a></li>
<li>Unraveling the Sense of Smell, Nobel Lecture, December 8, 2004: <a href="https://www.nobelprize.org/uploads/2018/06/buck-lecture.pdf">https://www.nobelprize.org/uploads/2018/06/buck-lecture.pdf</a></li>
<li>Buck L, Axel R. <em>Cell</em>. 1991;65(1):175-87. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/1840504/">1840504</a>.</li>
<li>Ressler KJ, et al. <em>Cell</em>. 1993;73(3):597-609. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/7683976/">7683976</a>.</li>
<li>Vassar R, et al. <em>Cell</em>. 1993;74(2):309-18. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/8343958/">8343958</a>.</li>
<li>Ressler KJ, et al. <em>Cell</em>. 1994;79(7):1245-55. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/7528109/">7528109.</a></li>
<li>Vassar R, et al. <em>Cell</em>. 1994;79(6):981-91. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/8001145/">8001145</a>.</li>
<li>Mombaerts P, et al. <em>Cell</em>. 1996;87(4):675-86. PMID: <a href="https://pubmed.ncbi.nlm.nih.gov/8929536/">8929536</a>.</li>
<li>National Smell and Taste Center: <a href="https://www.nidcd.nih.gov/research/labs/national-smell-taste-center">https://www.nidcd.nih.gov/research/labs/national-smell-taste-center</a></li>
</ol>
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