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Latest News

New grant to study AGO syndromes

The Independent Research Fund Denmark supports research on Argonaute patient mutations

New publication

New research shows that tiny timing errors in how AGO2 controls genes may explain brain changes in LESKRES syndrome

More on AGO Syndromes Conference 2025, 2024 and 2022.

What are AGO syndromes?

AGO syndromes are rare, neurodevelopmental, genetic disorders. Affected individuals may suffer from delayed motor development, seizures, problems speaking and understanding, and cognitive impairment.

The syndromes are associated with mutations in the genes AGO1 (NEDLBAS syndrome) and AGO2 (Lessel-Kreienkamp/Leskres syndrome). They may produce a similar, if not the same, syndrome.

We know little about these recently discovered, rare diseases, which brings no comfort to families. It is devastating to be told that the prognosis is unclear, that there is no treatment, and that there is not much that can be done for your child.

Our mission

We founded the AGO Alliance to:

find and fund paths to treatment,
connect families,
promote awareness and share information.

We believe in the potential of research and innovation to one day improve the life of not only our children but all children affected by AGO syndromes.

The Argonautes / AGO syndromes meeting is clearly the best for Argonaute scientists. It truly inspires us to push boundaries and accelerate our research. We’re here, dedicated to working for you, AGO families.

Prof. Simard

Professor at Centre de Recherche du CHU, Quebec

We felt very alone after the diagnosis, but the association and science committee gave us hope – to understand the disease, to come up with a treatment, and have the support of like-minded people.

Aldona

Mother and Science Officer

I'm impressed by how far the field is already pushing to understand the mutations after the first meeting with families.

Amy Buck

Professor at University of Edinburgh

To define ultra-rare genetic disorders we need a close interdisciplinary collaboration between patient families, clinicians, and diagnostic and research laboratories.

Prof. Lessel

Discoverer of AGO2/Leskres Syndrome