Global Collaboration Launches Streamlined Access to Shank3 cKO Research Model
In an important development for the field of neurodevelopmental disorders, a collaboration involving InnoSer, CureSHANK, and Ozgene has announced the introduction of a state-of-the-art Shank3 conditional knockout (cKO) mouse model. This innovative tool is poised to significantly advance research into Phelan-McDermid syndrome (PMS) and other disorders associated with SHANK3 haploinsufficiency. As researchers worldwide grapple with the complexities of these rare genetic conditions, this model is designed to streamline access to high-quality resources that can accelerate therapeutic development and scientific discovery.
The Shank3 cKO mouse model, engineered on the C57BL/6J background, provides researchers with a platform that encompasses precise genetic engineering capabilities. Notably, this model features an Exon 4-22 deletion of the Shank3 gene, achieved through the strategic implementation of loxP sites. Such precision allows for the excision of critical gene segments when employing Cre-driver lines, leading to a full knockout of Shank3. This genetic configuration not only facilitates the study of SHANK3 haploinsufficiency but also enables exploration of its implications on synaptic biology and behavioral manifestations in vivo.
Scientific literature identifies that the removal of exons 4-22 leads to a loss of all major murine SHANK3 isoforms. Therefore, researchers can expect to observe a range of behavioral, cognitive, and motor phenotypes characteristic of SHANK3-related conditions, which are pivotal for understanding the pathophysiology of PMS and autism spectrum disorder (ASD) related to SHANK3 genes. This model builds upon groundbreaking work conducted in previous studies, affirming its relevance in translational research.
Utilizing patented goGermline technology developed by Ozgene, the Shank3 cKO model promises enhanced genetic accuracy and reproducibility alongside improved ethical efficiencies in research practices. By establishing colonies in Indianapolis, USA, and offering additional housing solutions in Perth, Australia, Ozgene is positioning itself as the global distributor for this model, making it accessible to researchers across different geographical locations.
Dr. Frank Koentgen, founder of Ozgene, emphasized the significance of this model in providing a robust genetic platform for investigating disorders associated with SHANK3 deficiencies. As PMS and related disorders continue to pose considerable challenges in therapeutic development, tools that enable efficient research progression are essential for filling the gaps in our understanding and treatment of these complex conditions.
The Shank3 Exon 4-22 deletion model is tailored for a plethora of research applications, ensuring that its utility extends far beyond just basic characterization. Researchers can leverage this model for a variety of objectives, such as studying the details of synaptic biology and the mechanisms underlying SHANK3 haploinsufficiency. Furthermore, it opens avenues for the preclinical evaluation of innovative therapeutic approaches including gene therapies and antisense oligonucleotides (ASOs), which target the restoration of SHANK3 functionality.
In addition to this, the collaboration is preparing to launch a standardised preclinical testing platform specifically designed for Phelan-McDermid syndrome. This platform, officially set to debut in late 2026, will encompass a comprehensive suite of assessments ranging from biomarker analysis to sensorimotor and behavioral studies. These assessments are critical for therapeutic development initiatives, providing insights that pave the way for smoother translation from bench to bedside in drug development processes.
To facilitate the ordering process and improve accessibility, researchers can directly obtain the Shank3 Ex4-22 cKO model through Ozgene with various options available. This includes the provision of study-ready experimental cohorts, breeding pairs for internal use, and custom background backcrossing. Moreover, long-term management of colonies can be handled through Ozgene’s facilities either in Australia or the USA.
It is crucial to highlight that all transactions and distributions of these models are governed under standard use licenses. These licenses allow for internal research use and breeding yet prohibit onward distribution to third-party entities. This regulatory aspect ensures that researchers can utilize the models while respecting the intended ethical use framework established by the collaborating organizations.
The strategic collaboration between InnoSer, CureSHANK, and Ozgene marks a significant milestone in the research landscape for SHANK3-related disorders. By simplifying access to relevant genetic models and integrating complementary preclinical services, they foster a more efficient research environment. As a result, researchers are better equipped to navigate the complexities inherent in studying rare genetic disorders, ultimately hastening the process of drug discovery and innovative therapeutic solutions.
The Shank3 cKO model is not only set to transform individual research laboratories; it signifies a broader movement towards collaborative approaches in addressing pressing health challenges associated with neurodevelopmental disorders. With renewed optimism and tools that are both innovative and readily available, scientists can focus on what truly matters: the advancement of knowledge and therapeutic options for individuals living with disorders rooted in SHANK3 haploinsufficiency.
Subject of Research: Animals
Article Title: Global Collaboration Launches Streamlined Access to Shank3 cKO Research Model
News Publication Date: February 9, 2026
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Keywords: Shank3, Phelan-McDermid syndrome, neurodevelopmental disorders, gene therapy, research models, conditional knockout, Ozgene, InnoSer, CureSHANK
