CohortAutism research : official journal of the International Society for Autism Research2021

CIM6P/IGF-2 Receptor Ligands Reverse Deficits in Angelman Syndrome Model Mice.

Emmanuel Cruz, Giannina Descalzi, Adam Steinmetz and 3 others

PMID 33108069

WHAT IT FOUND

In mice with Angelman syndrome, a single injection of IGF-2 or M6P reversed memory, motor, and repetitive behavior deficits.

IGF-2 also reduced seizures. These findings are preclinical and have not been tested in humans.

Key findings

01Subcutaneous injection of IGF-2 or M6P reversed deficits in memory retention and flexibility in Angelman syndrome model mice.

02Both ligands reversed motor deficits, specifically hindlimb clasping and rotarod performance, with effects persisting for days.

03IGF-2, but not M6P, significantly decreased the frequency and severity of audiogenic seizures and reversed marble-burying impairments.

STILL TO COME

How it was doneWhat they found

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What it does not show

This is a preclinical study in mice; no human patients were involved. Only a single acute dose was tested, whereas human treatment would likely require chronic administration. The mechanisms of action for the observed behavioral improvements are not known. The study did not assess long-term safety or efficacy of repeated injections.

Declared interests

The study was supported by N.I.H. Extramural and Non-U.S. Gov't grants. No commercial conflicts of interest are reported in the provided text.

The easy way to misread this

Do not interpret these results as evidence that IGF-2 or M6P injections are effective treatments for Angelman syndrome in humans. The study was conducted exclusively in mice, and the safety and efficacy of these ligands in human patients have not been established.

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The study

Certainty of evidence
Low

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    Cite

    Emmanuel Cruz, Giannina Descalzi, Adam Steinmetz, et al. CIM6P/IGF-2 Receptor Ligands Reverse Deficits in Angelman Syndrome Model Mice. Autism research : official journal of the International Society for Autism Research. 2021.

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