Perlara’s Cure Odysseys

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Chinese Traditional Medicines are top hits for AFG3L2, a progressive neurodegenerative disease
We discovered a class of natural products called saponins in a yeast-powered drug repurposing screen for AFG3L2. Saponins are also rescuers of FARS2…
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Ethan O. Perlstein, Ph.D.
FARS2 Cure Odyssey
Wendy and David launched a yeast-powered drug repurposing project for their son Alexander, who has an ultra-rare mitochondrial disease. One of the top…
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Laura DeFrancesco
 and 
Ethan O. Perlstein, Ph.D.
KCNC1 drug repurposing screen
Eliana's V434L gain-of-function variant was expressed in Chinese Hamster Ovary cells and the Broad Repurposing Hub library was screened for channel…
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Ethan O. Perlstein, Ph.D.
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PACS2 drug repurposing pilot screen
A pilot screen of 1,411 compounds revealed 25 compounds that rescue Cell Painting phenotypes in PACS2 fibroblasts, including a hit that makes diseased…
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Ethan O. Perlstein, Ph.D.
Pharma Chameleon
Often hiding in plain sight, a small molecule can have multiple pharmacological identities. Context-switching and shape-shifting pharmacophores keep…
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Ethan O. Perlstein, Ph.D.
ADSL Cure Odyssey
A scientist and mother from Belgrade reached out last year about therapeutic options for her daughter suffering from an ultra-rare disease called ADSL…
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Ethan O. Perlstein, Ph.D.
FAMily ties
Less than three months ago, FAM177A1 Research Foundation hosted its first research roundtable. Drug repurposing and biomarker discovery projects are now…
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Ethan O. Perlstein, Ph.D.
Perlara’s Cure Odysseys
Stories from pioneering families battling genetic diseases at the frontier of precision medicine.

Perlara’s Cure Odysseys

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