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Icahn Genomics Institute

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to scan the genomes of healthy people age 30 and older who contribute their DNA to the effort with an initial focus on 127 diseases. Scientists anticipated that finding protective mechanisms for Mendelian diseases would be more straightforward than finding ones for complex or multifactorial diseases. Based on an analysis of publicly available data from 600,000 human genomes, scientists involved in the Resilience Project estimated that one person in 15,000 has a mechanism protecting against disease-causing genetic variants.
167:, biologists developing and testing new drugs and drug platforms, and data scientists working to identify causative agents of disease that can be targeted for therapy by building predictive models that better characterize disease. These models are constructed with multiple layers of biological data, including gene expression, metabolite, DNA, and protein information, and are combined with phenotypic and clinical data, predictive modeling, and probabilistic analysis to try to elucidate the complex mechanisms of disease. 138:. Its aim is to establish a new generation of medicines that can better treat diseases afflicting the world, including cancer, heart disease and infectious pathogens. To do this, the institute’s doctors and scientists are developing and employing new types of treatments that utilize DNA and RNA based therapies, such as 596:
Komiyama, Noboru H.; Collins, Mark O.; Fernández, Esperanza; Genovese, Giulio; Stahl, Eli; Duncan, Laramie; Kähler, Anna; Bergen, Sarah E.; Chambert, Kimberly; O’Dushlaine, Colm; Roussos, Panos; Solovieff, Nadia; Ruderfer, Douglas; Fromer, Menachem; Moran, Jennifer L.; Purcell, Shaun M. (1 February 2014).
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In 2014 the institute, in collaboration with Sage Bionetworks, announced a new project aiming to genotype up to 1 million people with the goal of identifying the rare biological mechanisms that keep people healthy when they have genetic variants that should cause disease. The Resilience Project aimed
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Also in 2022, Brian Brown's research published results of a study targeting solid tumors with chimeric antigen receptor (CAR)-T cell therapy, which had previously only been successful in blood cancers. The researchers engineered CAR T cells to target and destroy macrophages, an immune cell supporting
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in 2012 demonstrating the ability to derive enough information from non-DNA sources to identify individuals whose supposedly anonymized biological data is stored in large research databases. The authors reported that measuring RNA levels in tissue allowed them to infer a genetic barcode that could be
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Mouhieddine, Tarek H.; Van Oekelen, Oliver; Melnekoff, David T.; Li, Jeanne; Ghodke-Puranik, Yogita; Lancman, Guido; Thibaud, Santiago; Pan, Darren; Rajeeve, Sridevi; Agte, Sarita; Aleman, Adolfo; Sanchez, Larysa; Richard, Shambavi; Rossi, Adriana; Richter, Joshua; Cho, Hearn Jay; Rodriguez, Cesar;
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Sklar, Pamela; McCarroll, Steven A.; Sullivan, Patrick F.; Hultman, Christina M.; Lander, Eric S.; Scolnick, Edward M.; Gabriel, Stacey; Haggarty, Stephen J.; Grant, Seth G. N.; DePristo, Mark; Fennell, Tim; Garimella, Kiran; Shakir, Khalid; Banks, Eric; Magnusson, Patrik K. E.; Jyoti S. Choudhary;
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O’Donovan, Michael C.; Purcell, Shaun M.; Owen, Michael J.; Sklar, Pamela; Holmans, Peter; McCarroll, Steven A.; Palotie, Aarno; Kirov, George; Moran, Jennifer L.; Scolnick, Edward M.; Mahajan, Milind; Chambert, Kimberly; Rose, Samuel A.; Hannon, Eilis; Grant, Seth G.; Vihra Milanova; Banks, Eric;
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that explored the genetic complexity of schizophrenia. The exome sequencing studies of populations in Bulgaria and Sweden revealed that the disorder is likely caused by a lot of rare genetic mutations rather than a few common mutations. The projects also established the world’s largest database on
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Dhainaut, Maxime; Rose, Samuel; Akturk, Guray; Wroblewska, Aleksandra; Nielsen, Sebastian; Park, Eun; Buckup, Mark; Roudko, Vladimir; Pia, Luisanna; Sweeney, Robert; Le Berichel, Jessica; Wilk, C. Matthias; Bektesevic, Anela; Lee, Brian; Bhardwaj, Nina; Rahman, Adeeb; Baccarini, Alessia; Gnjatic,
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Dhainaut, Maxime; Rose, Samuel A.; Akturk, Guray; Wroblewska, Aleksandra; Nielsen, Sebastian R.; Park, Eun Sook; Buckup, Mark; Roudko, Vladimir; Pia, Luisanna; Sweeney, Robert; Le Berichel, Jessica; Wilk, C. Matthias; Bektesevic, Anela; Lee, Brian H.; Bhardwaj, Nina; Rahman, Adeeb H.; Baccarini,
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Campisi, Laura; Chizari, Shahab; Ho, Jessica S. Y.; Gromova, Anastasia; Arnold, Frederick J.; Mosca, Lorena; Mei, Xueyan; Fstkchyan, Yesai; Torre, Denis; Beharry, Cindy; Garcia-Forn, Marta; Jiménez-Alcázar, Miguel; Korobeynikov, Vladislav A.; Prazich, Jack; Fayad, Zahi A.; Seldin, Marcus M.; De
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paper, institute founding director Eric Schadt led a research team that utilized six different types of data (metabolite concentration, gene expression, DNA variation, DNA-protein binding, protein-metabolite interaction, and protein–protein interaction) to reconstruct networks involved in cell
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Barker, Douglas D.; Roussos, Panos; Johnson, Jessica S.; Humphreys, Isla; Carrera, Noa; Ruderfer, Douglas M.; Palta, Priit; Rees, Elliott; Georgieva, Lyudmila; Gormley, Padhraig; Dwyer, Sarah; Williams, Hywel J.; Kavanagh, David H.; Pocklington, Andrew J.; Fromer, Menachem (1 February 2014).
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The institute’s primary goal is to improve patient care through the use of gene, cell and nucleotide therapies. To achieve this goal, the Institute is formed of a cross-disciplinary mix of clinicians and scientists that include physicians treating patients with novel gene therapies in the
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used to match other materials to that same individual. This was noteworthy as validation of existing concerns among genomics scientists that it may not be possible to prevent the identification of an individual from genetic data even when that data is meant to be anonymous.
337:(ALS). The study found immune system dysfunction in patients and mice with ALS, and showed a high concentration of CD8 T cells in spinal cord and blood. The study was among the first publications to describe the immune system's involvement in neurodegeneration. 248:
reporting findings from a network-based study of late-onset Alzheimer's disease. The researchers constructed gene regulatory networks and discovered a neural structure involved in a pathway associated with onset of the disease.
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Former institute member Joel Dudley was named one of the 100 most creative people in business in a 2014 list compiled by Fast Company. The magazine said it chose Dudley "for splicing information with quality medical care."
154:, and searching for novel drug targets through the use of functional genomics and data science. The institute is led by Brian Brown, a leading expert in gene therapy, genetic engineering, and molecular immunology. 1049: 685:
Makiko Yasuda; Lin Gan; Brenden Chen; Senkottuvelan Kadirvel; Chunli Yu; John D Phillips; Maria I New; Abigail Liebow; Kevin Fitzgerald; William Querbes; Robert J Desnick (May 12, 2014).
367:, Michael Linderman, George Diaz, Ali Bashir, and Randi Zinberg taught the first class in which Mount Sinai medical students were able to fully sequence and analyze their own genomes. 1076: 1117:
Rubeis, Silvia; Bennett, Craig L.; Ostrow, Lyle W.; Lunetta, Christian; Squatrito, Massimo; Byun, Minji; Shneider, Neil A.; Jiang, Ning; La Spada, Albert R.; Marazzi, Ivan (2022).
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as the first therapy for acute hepatic porphyria, approved by the FDA in 2019. Institute researchers and clinicians also led the Phase 3 clinical trials for givosiran.
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Zhu, Jun; Sova, Pavel; Xu, Qiuwei; Dombek, Kenneth M.; Xu, Ethan Y.; Vu, Heather; Tu, Zhidong; Brem, Rachel B.; Bumgarner, Roger E.; Schadt, Eric E. (2012).
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The institute was formed in 2011 as the Institute for Genomics and Multiscale Biology, as part of Mount Sinai's Department of Genetics and Genomic Sciences.
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was named as founding director. The institute was renamed in 2012 when philanthropist Carl Icahn pledged $ 200 million to its parent organization, the
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https://aacrjournals.org/cancerimmunolres/article-abstract/10/11/1354/709883/Targeting-Macrophages-with-CAR-T-Cells-Delays?redirectedFrom=fulltext
382:. The center's goals included identification of biomarkers and drug targets for cancer and development of computational tools for drug discovery. 350: 131: 94: 1436: 744: 1396: 1303:
https://www.clinicalresearchnewsonline.com/news/2014/05/23/for-students-at-the-icahn-school-of-medicine-genomics-is-growing-more-personal
687:"RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice" 1510: 839: 463:"Stitching together Multiple Data Dimensions Reveals Interacting Metabolomic and Transcriptomic Networks That Modulate Cell Regulation" 1515: 1205: 1180: 1285: 285:
imaging technology called Perturb-map capable of identifying regulators in tumor microenvironments. The findings were published in
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Schadt, Eric E.; Woo, Sangsoon; Hao, Ke (2012). "Bayesian method to predict individual SNP genotypes from gene expression data".
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receptor, TGFbR, as regulators of two pathways significantly affecting tumor growth, architecture, and immune cell recruitment.
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tumor growth, in ovarian, lung, and pancreatic tumors in mice models, which successfully shrunk tumors and prolonged survival.
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In 2022, institute faculty Pei Wang and Avi Ma'ayan established a Proteogenomic Data Analysis Center funded by the
379: 315: 164: 1416: 653: 513:"Cell - Integrated Systems Approach Identifies Genetic Nodes and Networks in Late-Onset Alzheimer's Disease" 1314: 1371: 266:
In May 2014, institute faculty published proof-of-concept findings to support the clinical development of
36: 912:"Novel CRISPR Imaging Technology Reveals Genes Controlling Tumor Immunity | Mount Sinai - New York" 319: 1457:"CPTAC Announces New PCC, PGDAC, and PTRC Teams | Office of Cancer Clinical Proteomics Research" 1337: 698: 609: 551: 1401: 139: 63: 48: 1103:"New approach to immunotherapy provokes a robust anti-tumor immune response in preclinical models" 1063: 1031: 893: 793: 443: 1353: 1253: 1148: 1050:"Therapies That Engage T Cells Most Effective Against Relapsed or Refractory Multiple Myeloma" 1023: 971: 885: 785: 726: 635: 577: 494: 435: 271: 1421: 1345: 1243: 1166: 1138: 1130: 1013: 1003: 990:
Lagana, Alessandro; Moshier, Erin L.; Chari, Ajai; Jagannath, Sundar; Parekh, Samir (2022).
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In January 2014, scientists from the institute’s Division of Psychiatric Genomics including
179: 826:"Cell Papers on Viruses in Chinese Game Animals, Perturb-map, Tumor-Associated Macrophages" 1272:"Q&A: Mount Sinai's Milind Mahajan on Running a CLIA-Certified Genomics Core Facility" 1184: 322:(BiAbs) as a second-line treatment after a first-line immunotherapy treatment had failed. 301: 224: 1289: 1341: 1143: 1118: 1018: 991: 702: 613: 555: 966: 941: 880: 855: 721: 686: 630: 597: 572: 539: 489: 462: 289:
in 2022. Perturb-map was used in this study to identify the cytokine interferon gamma,
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patients. The study reported over an 80% response rate in patients treated with
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Institute faculty member Samir Parekh published results of a clinical trial in
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Alessia; Gnjatic, Sacha; Pe'Er, Dana; Merad, Miriam; Brown, Brian D. (2022).
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Manisha Balwani; Eliane Sardh; ENVISION Investigators (June 11, 2020).
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Sacha; Pe’er, Dana; Merad, Miriam; Brown, Brian (March 31, 2022).
672:"Schizophrenia could be caused by a wide variety of DNA mutations" 598:"A polygenic burden of rare disruptive mutations in schizophrenia" 329:
Institute faculty member Ivan Marazzi's research team published a
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Friend, S. H.; Schadt, E. E. (2014). "Clues from the resilient".
540:"De novo mutations in schizophrenia implicate synaptic networks" 290: 1397:""Genetic Heroes" May be Key to Treating Debilitating Diseases" 1375: 242:
In 2013, institute scientists published a paper in the journal
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In 2012, the institute received certification for the first
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is a biomedical and genomics research institute within the
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Biomedical and genomics research institute in New York, US
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paper in 2022 identifying the immune system's role in
306:(2022) focused on second-line treatments for relapsed 926:"Spatial CRISPR Genomics of Tumor Microenvironments" 840:"CRISPR Imaging Reveals Genes Behind Tumor Immunity" 812:"Spatial CRISPR Genomics of Tumor Microenvironments" 222:Scientists from the institute published a paper in 104: 88: 80: 72: 62: 42: 32: 24: 1181:"Partnering on Multiscale Biology - Bio-IT World" 281:Institute director, Brian Brown, developed a new 175:Research at the institute falls into six areas: 691:Proceedings of the National Academy of Sciences 8: 76:1425 Madison Avenue, New York, NY 10029-6501 19: 1437:"The Search for Genes That Prevent Disease" 1491:Genetics or genomics research institutions 18: 1247: 1142: 1017: 1007: 965: 879: 779: 720: 710: 629: 571: 488: 478: 1501:Icahn School of Medicine at Mount Sinai 1372:"Welcome | the Resilience Project" 391: 351:Icahn School of Medicine at Mount Sinai 132:Icahn School of Medicine at Mount Sinai 95:Icahn School of Medicine at Mount Sinai 7: 399: 397: 395: 256:published two papers in the journal 768:The New England Journal of Medicine 14: 1506:Organizations established in 2011 1496:Laboratories in the United States 1009:10.1182/bloodadvances.2022007923 274:which led to the development of 1167:"New ALS Research Breakthrough" 749:US Food and Drug Administration 99:Mount Sinai Hospital, New York 1: 335:amyotrophic lateral sclerosis 218:Notable academic publications 480:10.1371/journal.pbio.1001301 213:Therapeutic Target Discovery 210:Cancer Gene and Cell Therapy 1532: 1511:Systems science institutes 1135:10.1038/s41586-022-04844-5 958:10.1016/j.cell.2022.02.015 872:10.1016/j.cell.2022.02.015 207:and Microbial Therapeutics 1516:American medical research 380:National Cancer Institute 316:chimeric antigen receptor 314:redirection therapy with 165:Mount Sinai Health System 295:tumor growth factor beta 128:Icahn Genomics Institute 20:Icahn Genomics Institute 1350:10.1126/science.1255648 712:10.1073/pnas.1406228111 192:, and Cell Engineering 44:Field of research 37:Translational research 781:10.1056/NEJMoa1913147 320:bispecific antibodies 1292:on December 9, 2022. 1236:Nature Biotechnology 952:(7): 1223–1239.e20. 866:(7): 1223–1239.e20. 751:. November 20, 2019. 1402:Scientific American 1342:2014Sci...344..970F 1230:Schadt, E. (2012). 703:2014PNAS..111.7777Y 658:www.bio-itworld.com 622:10.1038/nature12975 614:2014Natur.506..185P 564:10.1038/nature12929 556:2014Natur.506..179F 118:/genomics-institute 21: 1105:. 25 October 2022. 1066:. 4 November 2022. 1052:. 9 November 2022. 363:Institute faculty 318:(CAR)-T cells and 270:therapy for acute 33:Research type 1336:(6187): 970–972. 1274:. 8 January 2013. 1129:(7916): 945–952. 774:(24): 2289–2301. 697:(21): 7777–7782. 608:(7487): 185–190. 550:(7487): 179–184. 272:hepatic porphyria 124: 123: 1523: 1475: 1474: 1467: 1461: 1460: 1453: 1447: 1446: 1433: 1427: 1426: 1422:Business Insider 1413: 1407: 1406: 1393: 1387: 1386: 1384: 1383: 1374:. Archived from 1368: 1362: 1361: 1325: 1319: 1318: 1311: 1305: 1300: 1294: 1293: 1288:. Archived from 1282: 1276: 1275: 1268: 1262: 1261: 1251: 1249:10.1038/nbt.2331 1227: 1221: 1220: 1218: 1217: 1202: 1196: 1195: 1193: 1192: 1183:. 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Index

Translational research
gene editing
gene therapy
genomics
Director
Affiliations
Icahn School of Medicine at Mount Sinai
Mount Sinai Hospital, New York
icahn.mssm.edu/research/genomics-institute
Icahn School of Medicine at Mount Sinai
New York City
CRISPR
siRNA
RNA vaccines
CAR T cells
Mount Sinai Health System
RNA Therapeutics
Nanomedicines
CRISPR
Asthma
allergies
Viral
Nature Genetics
PLoS Biology
Cell
Pamela Sklar
Nature
RNA interference
hepatic porphyria
givosiran

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