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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.

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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

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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.

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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

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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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Rilzabrutinib Cuts Flares in IgG4-Related Disease; Fast Track Granted in US

The Pharma Data

The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.

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Scientists develop technology that brings new precision to genome editing

Broad Institute

Choudhary is an assistant professor of medicine at Harvard Medical School The implications of this advance are wide-ranging. CRISPR-Cas9, often likened to “molecular scissors,” allows scientists to cut DNA at targeted sites to snip, repair, or replace genes.

DNA