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Researchers devise new way to target and correct disease-related proteins

Broad Institute

Related groups Xavier lab Over the past two decades, large genetic studies have linked tens of thousands of DNA variants to thousands of human traits and diseases. Yet, correcting the effects of those variants to treat disease has been hampered in part by the lack of precise molecular tools to do so.

Disease 139
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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My

Disease 80
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Scientists create next generation of tools in battle against brain disease

Broad Institute

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. The tools are known as enhancer AAV vectors.

Disease 72
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Count Me In launches new patient-partnered research project for translocation renal cell carcinoma

Broad Institute

The tRCC Project aims to address these challenges, by partnering directly with patients, caregivers, and advocates to deepen understanding of this rare disease and generate new insights that could lead to new treatments. Tags: Count Me In Cancer Rare Disease

Research 113
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Feng Zhang awarded the National Medal of Technology and Innovation

Broad Institute

Two Broad affiliates also received the award: Paula Hammond, an MIT Institute Professor and vice provost for faculty, and David Walt, Hansjrg Wyss Professor of Biologically Inspired Engineering at Harvard Medical School and a professor of pathology at Brigham and Womens Hospital. Lisa Yang and Hock E.

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Scientists link certain gut bacteria to lower heart disease risk

Broad Institute

Scientists link certain gut bacteria to lower heart disease risk By Allessandra DiCorato April 2, 2024 Breadcrumb Home Scientists link certain gut bacteria to lower heart disease risk Study finds several species of cholesterol-metabolizing bacteria in people with lower cholesterol levels. Online April 2, 2024. DOI:10.1016/j.cell.2024.03.014.

Disease 145
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Researchers map communities of single cells in metastatic breast cancers

Broad Institute

Researchers have typically analyzed these cells as a mixed-together group, but this approach can miss rare cell types, and makes it difficult to draw conclusions about how cells interact to drive the disease. They also applied four spatial transcriptomics methods, including Slide-Seq and MERFISH, to a subset of the biopsies.

Research 127