Research Event Encourages Academic Cooperation and Progress at Batten Institute
**University of Rochester at the Forefront of CLN3 Batten Disease Research and Care**
The University of Rochester Medical Center is making significant strides in the field of CLN3 Batten disease and other neuronal ceroid lipofuscinosis (NCL) disorders. As one of the leading institutions in this area, the university is dedicated to fostering collaboration and growing understanding of these conditions to improve care.
In a recent event, basic scientists and clinical researchers from across the university gathered to discuss the latest research on CLN3 Batten disease. The University of Rochester Intellectual and Developmental Research Center (UR-IDDRC) and the University of Rochester Batten Center (URBC) hosted a Batten Research Day in March, which brought together key stakeholders to share insights and advance knowledge about the disease.
CLN3 Batten disease primarily affects children, leading to severe neurological and visual impairments, motor and cognitive decline, seizures, and premature death. The disease is caused by mutations in the CLN3 gene, and current research often involves using human pluripotent stem cells to model the disease and explore potential treatments, such as gene therapies or drugs targeting specific cellular pathways.
One of the potential areas of research at universities like the University of Rochester is stem cell research. By utilizing stem cells, researchers can study disease progression and screen potential treatments more effectively. Another focus area is gene therapy, where researchers are working to develop methods to correct the genetic defect causing the disease. Clinical trials are also crucial, as they allow for the evaluation of the safety and efficacy of new treatments.
The University of Rochester's work on CLN3 Batten disease and other NCLs is multifaceted, encompassing both research and treatment. In addition to their efforts in research, the university is also actively involved in conducting clinical trials to evaluate the safety and efficacy of new treatments.
Moreover, the university's commitment to involving families in the research process is evident. John Foxe, PhD, director of the Del Monte Institute for Neuroscience and co-director of the UR-IDDRC, emphasized the importance of having families involved in the research process to grow understanding of the disease and improve care. Parents of children with CLN3 Batten disease and Amy Fenton Parker, president and CEO of the Batten Disease Support, Research, and Advocacy Foundation (BDSRA), participated in the research day, offering firsthand experiences and insight into the lived experiences of children and families impacted by Batten disease.
The University of Rochester has been designated as a Center of Excellence by the BDSRA for its work on CLN3 Batten disease and other NCLs. In 2020, the National Institute of Child Health and Human Development (NICHD) designated the University as an Intellectual and Developmental Disability Research Center (IDDRC), making the disease the Center's principal project. The university is also one of eight institutions with the trifecta of federal awards related to Intellectual and Developmental Disabilities (IDDs). Along with the IDDRC, the University has a long-standing status as a University Center of Excellence in Developmental Disabilities Education, Research, and Service (UCEDD) and Leadership Education in Neurodevelopmental and Related Disabilities (LEND).
For the most accurate and up-to-date information about research and care at the University of Rochester, it is best to contact the university directly or visit their medical or research department websites. The university's focus on CLN3 Batten disease and other NCLs is driven by a commitment to fostering collaboration and growing understanding of these conditions to improve care for affected children and families.
- The University of Rochester's research extends beyond CLN3 Batten disease, also focusing on various other medical-conditions such as chronic-diseases, cancer, and mental-health, with a particular interest in neurological-disorders.
- In line with their commitment to health-and-wellness, the University of Rochester conducts research on developing treatments for diseases like CLN3 Batten, including the use of stem cells, gene therapies, and drug therapies targeting specific cellular pathways.
- By involving families in the research process, the University of Rochester aims to ensure that their work on conditions like CLN3 Batten disease aligns with the needs and concerns of those directly affected, maintaining a focus on improving care for children and families impacted by such medical-conditions.
