mouse model

June 15, 2018June 15, 2018

New Mouse Model of Late Infantile Batten Disease May Help Assess Potential Therapies

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A new mouse model of late infantile Batten disease may help to efficiently investigate personalized therapies, a study suggests. The research, “A tailored mouse model of CLN2 disease: A nonsense ... Read more

May 22, 2018May 22, 2018

Protein in Specific Cells Involved in Batten Disease Neurodegeneration, Mouse Study Suggests

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Therapeutic approaches that target production of a protein called progranulin in specific brain cells may help preserve brain function in Batten disease, according to a mouse study. The study, “Conditional loss of progranulin ... Read more

April 24, 2018April 24, 2018

Several Compounds Found to Correct Brain Defects in Mouse Model of CLN3 Disease

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Several compounds were able to correct defects in the brain caused by juvenile neuronal ceroid lipofuscinosis (CLN3) in both cellular and mouse models, uncovering potential therapeutic candidates for treating the disease. Because ... Read more

April 10, 2018April 10, 2018

Gene Therapy Prevents Vision Loss Caused by CLN6 Disease in Mouse Study

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Gene therapy directly targeting a specific type of neuron in the retina of the eye was able to prevent vision loss in late infantile Batten disease caused by rare CLN6 ... Read more

March 16, 2018March 16, 2018

New Mouse Model and Gene-Editing Tool May Help Advance CLN2 Research

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A genetically modified mouse with increased levels of a specific enzyme in the brain, along with a new genetic tool, may help researchers further their understanding of late infantile neuronal ... Read more

February 23, 2018March 23, 2018

Metallothioneins Show Promise as Therapeutic Targets in Mouse Model of Batten Disease

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A family of enzymes called metallothioneins potentially could be new therapeutic agents in the treatment of lysosomal storage disorders (LSDs), including Batten disease, a mouse model shows. The study, ... Read more

December 5, 2017

Mouse Model Helps Researchers Identify Processes Underlying Batten Disease Symptoms

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Researchers found, in a new mouse model for Batten disease, that defective synapses in specific brain regions may be the cause of disease symptoms. The study “Defective synaptic transmission ... Read more

October 24, 2017October 24, 2017

Brain Cell Function Impaired in Early Forms of Batten Disease, Mouse Study Shows

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Glial cell function is impaired in the juvenile form of Batten disease, leading to nerve cell loss, a mouse study has found. The study, “Glial cells are functionally impaired ... Read more

May 23, 2017

Lopid Increases Longevity in Mice with Late Infantile NCL, Study Finds

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Lopid (gemfibrozil), an FDA-approved lipid-lowering drug, was found to improve life expectancy and motor function in mice with late infantile neuronal ceroid lipofuscinosis (late infantile NCL, or LINCL), which belongs ... Read more

May 11, 2017May 11, 2017

Experiments Indicate Gene Therapy for Batten Should Aim for Low Levels of Activation

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Mouse experiments with a gene therapy for juvenile Batten disease indicate that lower activation of the mutated CLN3 gene are more beneficial than high levels. While the research team ... Read more

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