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Cognition and behavior in neurofibromatosis type 1: report and perspective from the Cognition and Behavior in NF1 (CABIN) Task Force
Journal article   Open access   Peer reviewed

Cognition and behavior in neurofibromatosis type 1: report and perspective from the Cognition and Behavior in NF1 (CABIN) Task Force

David H Gutmann, Corina Anastasaki, Aditi Gupta, Yang Hou, Stephanie M Morris, Jonathan M Payne, Jacob Raber, Seth M Tomchik, Linda Van Aelst, James A Walker, …
Genes & development, Vol.39(9-10), pp.541-554
05/01/2025
DOI: 10.1101/gad.352629.125
PMCID: PMC12047663
PMID: 40127956
url
https://doi.org/10.1101/gad.352629.125View
Published (Version of record) Open Access

Abstract

Individuals with neurofibromatosis type 1 (NF1) are prone to the evolution of neurodevelopmental symptomatology including motor delays, learning disabilities, autism, and attention deficits. Caused by heterozygous germline mutations in the gene, this monogenic condition offers unique opportunities to study the genetic etiologies for neurodevelopmental disorders and the mechanisms that underlie their formation. Although numerous small animal models have been generated to elucidate the causes of these alterations, there is little consensus on how to align preclinical observations with clinical outcomes, harmonize findings across species, and consolidate these insights to chart a cohesive path forward. Capitalizing on expertise from clinicians; human, animal, and cellular model research scientists; and bioinformatics researchers, the first Cognition and Behavior in NF1 (CABIN) meeting was convened at the Banbury Center of Cold Spring Harbor Laboratory in October 2024. This Perspective summarizes the state of our understanding and a proposed plan for future investigation and exploration to improve the quality of life of those with NF1.
Informatics Autism clinical translation cognition NF1 preclinical modeling attention deficit

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