Masayuki Yazawa
Icahn School of Medicine at Mount Sinai, NY·Pharmacology
A faulty calcium channel in heart muscle cells can throw off the heartbeat and cause dangerous arrhythmias. The same lab also builds new genetic tools in mice and uses them to track blood stem cells.
- Fields
Pharmacology
Medicine & Clinical Research
Genetics
- Topics
Timothy syndrome
long QT syndrome
cardiac arrhythmia
- Grants
- 2 known grants, through Jun 2027
What they work on
- Design, synthesize and characterize new small-molecule Sigma 1 receptor agonists that relieve Timothy syndrome heart cell defects, testing them in human iPSC-derived heart muscle cells and rodent models.
- Test whether the new Sigma 1 receptor agonists also correct defects in common genetic long QT syndromes types 1 and 2.
- Develop new mouse genetic tools that improve the precision in space and time of Cre recombinase and Cas9-based gene knockout and lineage tracing.
- Apply the new tools to trace hematopoietic stem cells clone by clone in living mice, to learn how the adult bone marrow stem cell pool is established.
More about the research
Its two projects therefore pair drug discovery for a rare inherited heart disorder with technology for following individual stem cells in a living animal.
Why it matters
Timothy syndrome and other genetic long QT syndromes cause life-threatening arrhythmias with limited treatment options. New Sigma 1 receptor agonists could become drug candidates for these patients. Understanding how the adult blood stem cell pool forms could inform bone marrow transplantation, and the new genetic tools may be useful in many other research fields.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
David Auerbach
Upstate Medical University, NY·Physiology
Test whether rabbits with a CRISPR-made Kcnh2 pore mutation reproduce the brain and heart problems of LQT2 patients with epilepsy, including QT prolongation, spontaneous seizures and seizure-related sudden death.
Anita Saraf
University of Pittsburgh, PA·Medicine & Clinical Research·Early-career
Determine how inflammatory cytokines, which stay chronically elevated in single-ventricle patients, change calcium handling and contraction in iPSC-derived heart muscle cells carrying hypomorphic NOTCH1 mutations from HLHS patients.
Richard Z Lin
Stony Brook University, NY·Pharmacology
Identify compounds that increase the IKs potassium current by acting on its calmodulin and ATP interactions or on pore opening, using structure-based computer screening of a library of more than one million compounds.
The school
Icahn School of Medicine at Mount Sinai: 45 labs on PI Pages, $1.0B a year on research, 62 research doctorates a year.
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