Perlara’s Cure Odysseys
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FOXG1 Cure Odyssey
A mother’s “darkest days” leads to a foundation and a magazine dedicated to families caring for children with rare diseases.
Feb 29
•
Laura DeFrancesco
and
Ethan O. Perlstein, Ph.D.
2
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FOXG1 Cure Odyssey
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SURF1 ReFRAME recap
On behalf of Cure Mito Foundation, last week we completed a comprehensive 13,500-compound drug repurposing screen using a Leigh Syndrome yeast avatar of…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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SURF1 ReFRAME recap
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SURF1 yeast hits validated in patient fibroblasts
Cure Mito Foundation partnered with Perlara on yeast-powered drug repurposing screens for SURF1 Leigh Syndrome. We found three compounds that increase…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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SURF1 yeast hits validated in patient fibroblasts
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Drug repurposing with yeast avatars of SURF1 Leigh Syndrome
We started working with Cure Mito in August on a yeast drug repurposing project based on the template of our previous work with UCSF on MEPAN. Last week…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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Drug repurposing with yeast avatars of SURF1 Leigh Syndrome
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Yeast-powered drug repurposing screens for SURF1 Leigh Syndrome are done 💊♻️ ✅
On behalf of Cure Mito Foundation, we completed high-throughput drug screens and dose response studies in yeast. Next up: validating hits in SURF1…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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Yeast-powered drug repurposing screens for SURF1 Leigh Syndrome are done 💊♻️ ✅
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Leigh Syndrome drug repurposing updates
In collaboration with Cure Mito Foundation and using a SURF1-deficient yeast model, we're validating hits from the pilot screen, and we just screened a…
Feb 5
•
Ethan O. Perlstein, Ph.D.
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Leigh Syndrome drug repurposing updates
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Unexpected PLOD1 twist
Collagen is a slippery eel. After trying and failing to visualize collagen inside PLOD1-kEDS fibroblasts, we pivoted to measuring collagen secreted by…
Jan 31
•
Ethan O. Perlstein, Ph.D.
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Unexpected PLOD1 twist
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The Anti-Portfolio of Pharma
The unlikely intersection of overlooked diseases, overlooked assets and overlooked founders is the place we've called home for the past 10 years. When…
Jan 7
•
Ethan O. Perlstein, Ph.D.
8
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The Anti-Portfolio of Pharma
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Perlara’s Cure Odysseys
Stories from pioneering families battling genetic diseases at the frontier of precision medicine.
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