Research

Hormonal

DL001 effectively suppresses hyperactive mTORC1 signaling in cells lacking a functional TSC (Tuberous Sclerosis Complex), restoring normal gene expression.

DL001 is shown to be as effective as rapamycin in suppressing the overactive mTORC1 signaling found in Tuberous Sclerosis Complex (TSC) cells. This suggests DL001 could be a safer alternative for treating TSC, as it may avoid the metabolic side effects associated with current rapalog treatments, although this is currently limited to cell culture models.

GoodSupportsHIGH confidence
We find that MEFs lacking Tsc1 have increased expression of a number of genes involved in metabolic pathways... and that the expression of these genes can be repressed to normal levels by treatment with either 100 nM rapamycin or 100 nM DL001.
Katherine H. Schreiber et al. · Nature Communications · 2019

Why this rating

Strong in vitro data showing gene expression normalization.

Source

A novel rapamycin analog is highly selective for mTORC1 in vivo

Katherine H. Schreiber et al. · Nature Communications · 2019

DOI 10.1038/s41467-019-11174-0

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DOI resolved against Crossref · corpus check 2026-06-10

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