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Stuart L. Schreiber
Broad Institute
132Publications
49H-index
9,747Citations
Publications 136
Newest
#1Karolina Michalska (U of C: University of Chicago)H-Index: 12
#2Changsoo Chang (U of C: University of Chicago)H-Index: 1
Last.Andrzej Joachimiak (U of C: University of Chicago)H-Index: 64
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#2Eric MinikelH-Index: 12
Last.Sonia M. VallabhH-Index: 4
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Antisense oligonucleotides (ASOs) designed to lower prion protein (PrP) expression in the brain through RNase H1-mediated degradation of PrP RNA are in development as prion disease therapeutics. ASOs were previously reported to sequence-independently interact with PrP and inhibit prion accumulation in cell culture, yet in vivo studies using a new generation of ASOs found that only PrP-lowering sequences were effective at extending survival. Cerebrospinal fluid (CSF) PrP has been proposed as a ph...
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#1Eric MinikelH-Index: 12
#2Eric KuhnH-Index: 16
Last.Steven A. CarrH-Index: 18
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2 CitationsSource
#1Timothy A. Lewis (Broad Institute)H-Index: 15
#2Luc de Waal (Broad Institute)H-Index: 3
Last.Matthew Meyerson (Broad Institute)H-Index: 160
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6-(4-(Diethylamino)-3-nitrophenyl)-5-methyl-4,5-dihydropyridazin-3(2H)-one, or DNMDP, potently and selectively inhibits phosphodiesterases 3A and 3B (PDE3A and PDE3B) and kills cancer cells by inducing PDE3A/B interactions with SFLN12. The structure-activity relationship (SAR) of DNMDP analogs was evaluated using a phenotypic viability assay, resulting in several compounds with suitable pharmacokinetic properties for in vivo analysis. One of these compounds, BRD9500, was active in an SK-MEL-3 xe...
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#1Andrew G Reidenbach (Broad Institute)H-Index: 1
#2Eric Minikel (Broad Institute)H-Index: 12
Last.Sonia M. Vallabh (Broad Institute)H-Index: 4
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Antisense oligonucleotides (ASOs) designed to lower prion protein (PrP) expression in the brain through RNAse H1-mediated degradation of PrP RNA are in development as prion disease therapeutics. ASOs were previously reported to sequence-independently interact with PrP and inhibit prion accumulation in cell culture, yet in vivo studies using a new generation of ASOs found that only PrP-lowering sequences were effective at extending survival. Cerebrospinal fluid (CSF) PrP has been proposed as a ph...
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#1Eric Vallabh Minikel (Broad Institute)H-Index: 4
#2Konrad J. Karczewski (Broad Institute)H-Index: 35
Last.Daniel G. MacArthur (Broad Institute)H-Index: 57
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Human genetics has informed the clinical development of new drugs, and is beginning to influence the selection of new drug targets. Large-scale DNA sequencing studies have created a catalogue of naturally occurring genetic variants predicted to cause loss of function in human genes, which in principle should provide powerful in vivo models of human genetic "knockouts" to complement model organism knockout studies and inform drug development. Here, we consider the use of predicted loss-of-functio...
5 CitationsSource
#1Sonia M. Vallabh (Broad Institute)H-Index: 4
#2Chloe K. Nobuhara (Harvard University)H-Index: 6
Last.Stuart L. Schreiber (Broad Institute)H-Index: 49
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Reduction of native prion protein (PrP) levels in the brain is an attractive strategy for the treatment or prevention of human prion disease. Clinical development of any PrP-reducing therapeutic will require an appropriate pharmacodynamic biomarker: a practical and robust method for quantifying PrP, and reliably demonstrating its reduction in the central nervous system (CNS) of a living patient. Here we evaluate the potential of ELISA-based quantification of human PrP in human cerebrospinal flui...
5 CitationsSource
#1Eric Minikel (Broad Institute)H-Index: 12
#2Eric Kuhn (Broad Institute)H-Index: 16
Last.Steven A. Carr (Broad Institute)H-Index: 18
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Therapies currently in preclinical development for prion disease seek to lower prion protein (PrP) expression in the brain. Trials of such therapies are likely to rely on quantification of PrP in cerebrospinal fluid (CSF) as a pharmacodynamic biomarker and possibly as a trial endpoint. Studies using PrP ELISA kits have reproducibly shown that CSF PrP is lowered in the symptomatic phase of disease, a potential confounder for reading out the effect of PrP-lowering drugs in symptomatic patients. To...
1 CitationsSource
#1Andrew L. Hong (Broad Institute)H-Index: 8
#2Yuen-Yi Tseng (Broad Institute)H-Index: 6
Last.William C. Hahn (Broad Institute)H-Index: 108
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Renal medullary carcinoma (RMC for short) is a rare type of kidney cancer that affects teenagers and young adults. These patients are usually of African descent and carry one of the two genetic changes that cause sickle cell anemia. RMC is an aggressive disease without effective treatments and patients survive, on average, for only six to eight months after their diagnosis. Recent genetic studies found that most RMC cells have mutations that prevent them from producing a protein called SMARCB1. ...
1 CitationsSource
#1Andrew L. Hong (Broad Institute)H-Index: 8
#2Yuen-Yi Tseng (Broad Institute)H-Index: 6
Last.William C. Hahn (Broad Institute)H-Index: 108
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