TPG Projects

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MCL1

MCL1 is a highly attractive therapeutic target in cancer. It is a specialized gene that interrupts programmed cell death, is commonly altered in tumor cells, and may aid tumor development as well as resistance to chemotherapy. Using a variety of innovative approaches, Broad Institute scientists have begun to lay the necessary groundwork to design transformative therapeutic strategies for the MCL1 pathway.
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PCSK9

Coronary artery disease is one of the leading causes of death worldwide. Recent research has shown that the PCSK9 gene regulates cholesterol levels in the blood, and is one of the key genes that drives elevated levels of LDL cholesterol. A therapy that successfully targets the PCSK9 protein and modulates LDL cholesterol would transform the treatment of coronary artery disease.
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ERG

In more than half of all prostate cancers, two genes, normally separate, become fused. This causes one of the genes, called ERG, to become permanently turned on, constantly signaling cancer cells to grow and invade. Broad Institute scientists are pursuing a comprehensive and innovative drug discovery program aimed at ERG, a challenging but promising drug target.
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BCL11A

Evidence suggests that BCL11A transcriptionally represses fetal hemoglobin (HbF) in adults. Increased HbF ameliorates the major globin disorders, sickle cell disease (SCD) and β-thalassemias. Broad Institute scientists are pursuing several lines of investigation to identify small molecule inhibitors of BCL11A function for therapeutic HbF reactivation.
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MUC1

Medullary cystic kidney disease type 1 (MCKD1) is a rare disorder characterized by autosomal dominant inheritance of tubulo-interstitial kidney disease. Recent studies have identified mutations in the mucin 1 (MUC1) gene as causal for this disease. Broad researchers have initiated a project aimed at modifying disease progression in affected MCKD1 patients. MUC1 transcription, targeted degradation of mutant MUC1 protein, and the potential for gene therapy will be evaluated as part of the therapeutic strategy
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FadD32

FadD32 is an essential enzyme for biosynthesis of mycolic acids, which comprise an integral component of the cell envelope in Mycobacterium tuberculosis. Broad scientists have recently discovered potent inhibitors of FadD32 using a full arsenal of genetic, protein, and small-molecule tools to identify new treatment modalities. These efforts have uncovered additional opportunities to inhibit M. tuberculosis during both its replicating and non-replicating phases. This “dual-hit” approach may improve treatment outcomes especially versus drug-resistant strains.