Broad Institute of MIT and Harvard

Congratulations to Broad core institute member Feng Zhang on receiving the National Medal of Technology and Innovation, the nation’s most prestigious honor for American innovators! Zhang is widely recognized for his pioneering work in developing the CRISPR-Cas9 genome-editing system and its use in eukaryotic cells. This and other molecular tools developed by Zhang have accelerated biomedical research worldwide.

Scientists need new ways to target disease-related genetic variants and correct their effects in cells. A research team led by Ramnik Xavier, Stuart Schreiber, and Tan Zher Yin at the Broad, Massachusetts General Hospital, and Harvard University built a remarkably diverse and massive collection of molecular compounds known as chemical inducers of proximity (CIPs), designed to bring two proteins together and use one as a shield to stabilize the other and reverse its disease-causing effects. From their library of 3 million compounds, the team identified one that targets a protein altered in some patients with Crohn’s disease and showed that it can reverse the variant’s detrimental effects on the cell. The approach could potentially be used to recruit proteins with other functional effects or target disease risk factors in only certain cell types or tissues. #BroadInstitute #Science #ScienceNews #Research #ScientificResearch #CrohnsDisease

In the Novo Nordisk Foundation Center Broad Institute of MIT and Harvard we are really excited to host the 31st Novo Nordisk Foundation Science Cluster Conference 'Genomic Mechanisms of Disease'. Recent years have seen tremendous progress in mapping the genetic basis of common diseases revealing remarkable complexity and posing substantial challenges in the interpretation of these results. To address this gap, new approaches have been pioneered to develop catalogs of multimodal single-cell data; faithful cell models that recapitulate disease-relevant biology; technologies for genome-scale perturbations; and powerful new ML/AI approaches for interpreting and predicting biology. One of the grand challenges on the frontier of human genomics and health is to develop paradigms that build on these advances to scale the understanding of genomic mechanisms driving complex disorders. This will enable a deep understanding of the biological programs affected in diseases, and lay the foundation for preventive strategies, genomic medicine, and rationally designed therapeutics to benefit patients and societies across the planet. This conference will bring together a vibrant global community of thought leaders in: -Epigenetics and gene regulation -Medical and population genetics -Variant to function: revealing disease-relevant biology -Genomic medicine -Foundation AI models of biology -Translating genomic mechanisms to therapeutics We have a stellar lineup of confirmed speakers including: Daphne Koller (insitro), @Jonathan Pritchard (Stanford University), Wendy Bickmore (The University of Edinburgh), Alex Meissner (Max Planck Institute for Molecular Genetics), Vadim Gladyshev (Harvard University), Mark McCarthy (Genentech), Claudia Langenberg (Queen Mary University of London), Eimear Kenny (Institute for Genomic Health Mount Sinai), Gosia Trynka (Wellcome Sanger Institute), ANSHUL KUNDAJE (Stanford University), Marcelo Nobrega (University of Chicago), Oliver Stegle (EMBL), Caroline Uhler (Broad Institute of MIT and Harvard / Massachusetts Institute of Technology), Mitchell Lazar (University of Pennsylvania), Peter Donnelly (University of Oxford and GENOMICS PLC). Thank you to the organizing committee: Melina Claussnitzer, Bradley Bernstein, Benjamin Neale, Simon Rasmussen, Ruth Loos, Liz Gaskell, Annika Weimer-Lindeboom and to the Novo Nordisk Foundation for generous support and logistical assistance. Please join us for an exciting interactive meeting of science and discussions:

Congratulations to the Broad Institute on 20 years of advancing science and improving human health through groundbreaking biomedical research. These inspiring remarks from our co-founder Edye Broad reflect our collective belief in the power of science to never stop pushing for the kind of change that makes the world a better place.

This year, the Broad Institute has celebrated our 20th anniversary – two decades of advancing human health through science. As we conclude this milestone celebration, we are honored to share a special video from our co-founder, Edythe Broad. We are deeply grateful to Eli and Edythe Broad for their extraordinary vision, steadfast support, and lasting partnership that have empowered the Broad Institute’s science and community. #BroadInstitute The Eli and Edythe Broad Foundation #Broadat20

Genetics contributes to dilated cardiomyopathy (DCM) — where the heart muscle enlarges and weakens — but the mechanisms linking DNA and disease have been unclear. An international team led at Broad by Sean Joseph Jurgens, Joel Rämö, Leonoor Wijdeveld, Mark Chaffin, Liam Gaziano, Aarno Palotie, Patrick Ellinor, Mark Daly, and Krishna Aragam conducted a multi-cohort GWAS including more than 9,300 DCM cases and 946,000 controls, finding 70 regions of the genome and 63 genes associated with DCM. Deeper analysis suggested that cardiomyocytes play a central role in disease, and showed potentially actionable connections between increased body weight, high blood pressure, and DCM risk. Learn more in Nature Genetics. #BroadInstitute #Science #ScienceNews #Research #ScientificResearch

Hypoxia may one day have a role in treating mitochondrial diseases like Leigh syndrome and Friedrich's ataxia, especially if researchers can develop a way of safely inducing hypoxia pharmaceutically. In the Journal of Clinical Investigation, Hong Wang, Vamsi Mootha, and colleagues describe a preclinical proof-of-concept mouse study of a "hypoxia-in-a-pill" regimen involving two FDA-approved drugs. The combination reduced brain tissue oxygen to levels comparable to breathing air at 11 percent O2 (as opposed to the normal 21 percent). Healthy mice tolerated the regimen well, and those carrying Leigh syndrome-associated mutations lived longer and showed improvement in neurological disease. Learn more: https://lnkd.in/eRiVW7Fq #BroadInstitute #Science #ScienceNews #Research #ScientificResearch

🔬 CHBV Co-op Spotlight! 🔬 Madelynn Coe is an undergraduate co-op researcher in her fourth year at Northeastern University. She is pursuing a degree in behavioral neuroscience with a minor in data science. Prior to the Broad, she worked at AbbVie on their Parkinson’s Histology team.

🔬 CHBV Co-op Spotlight! 🔬 Riyaan Gunaratne is an undergraduate co-op researcher and fourth year behavioral neuroscience major at Northeastern University with a minor in sociology. Riyaan hopes to further his career in neuroscience by earning his MD-PhD and becoming the PI of his own lab.

To better understand the host-pathogen interactions underlying tuberculosis (TB), Yang Luo and Soumya Raychaudhuri teamed up with Brigham and Women's Hospital's Chuan-Chin Huang and Megan Murray; the Harvard T.H. Chan School of Public Health's Nicole Howard, Xin Wang, Qingyun Liu, and Sarah Fortune; and colleagues to study host and Mycobacterium tuberculosis (Mtb) genomes from TB patients in Peru. Their findings, published in Nature Communications, reveal an association between a noncoding variant in the human FLOT1 gene and an Mtb lineage with an altered metabolic state, but also highlight the dynamic and complex interplay of host, pathogen, and environmental factors that shape infectious diseases. #BroadInstitute #Science #ScienceNews #Research #ScientificResearch