OtherMolecular autism2017

A brain proteomic investigation of rapamycin effects in the Tsc1+/- mouse model.

Hendrik Wesseling, Ype Elgersma, Sabine Bahn

PMID 28775826

WHAT IT FOUND

In mice modelling tuberous sclerosis, rapamycin changed hippocampal proteins linked to translation and myelin.

The work maps molecular targets, not patient symptoms or therapy outcomes.

Key findings

0151 proteins were altered in the frontal cortex and 108 in the hippocampus of Tsc1+/− mice compared with wildtype mice.

02In the hippocampus, rapamycin changed 231 proteins in mutant mice and 106 in wildtype mice.

03Rapamycin normalized 41 proteins altered in untreated mutant mice: 33 to wildtype levels and 8 to below or above baseline.

STILL TO COME

How it was doneWhat they found

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What it does not show

The study was done in mice, not in people receiving therapy. It measured protein abundance, not speech, movement, cognition, or daily function. The mouse model did not show typical human brain pathology such as seizures or lesions. The supplied text does not state the total number of mice analysed. Rapamycin was tested in mice, so the findings cannot be used to decide whether to treat a patient.

Declared interests

Funding was from Stanley Medical Research Institute. The supplied text does not state other conflicts.

The easy way to misread this

Do not read this as evidence that rapamycin improves therapy outcomes for people with tuberous sclerosis or autism. It measured protein changes in mouse brain tissue and did not test a clinical outcome in people.

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The study

Certainty of evidence
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    Hendrik Wesseling, Ype Elgersma, Sabine Bahn A brain proteomic investigation of rapamycin effects in the Tsc1+/- mouse model. Molecular autism. 2017.

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