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A new international consortium to promote Stem Cell-Based Therapy for Huntington s disease

The Stem Cells for Huntington’s Disease (SC4HD) is a new international consortium created to promote  advanced therapy medicinal product (ATMP) through cell transplantation studies on Huntington’s Disease. The entity, made up of twenty-eight renowned researchers from ten countries, has been officially presented in a recent publication in the  J ournal of Huntington’s Disease. The main goals of SC4HD researchers are to work together with the HD community, develop criteria and guidance and address the main challenges to rapidly and safely bring potentially beneficial stem cell-based therapies to patients with this devastating disease.  According to Josep M. Canals, director of Creatio and member of the steering committee of SC4HD, “there is an exciting opportunity to apply stem cell-based therapies in Huntington’s disease (HD), a devastating neurodegenerative disease that mainly affects neurons in the brain nucleus known as striatum”. 

New therapeutic target for Huntington s treatment

 E-Mail IMAGE: From left to right: Mercè Masana, Ana Gámez-Valero, Eulàlia Martí, Anna Guisado-Corcoll, Esther Pérez-Navarro and Maria Solaguren-Beascoa. view more  Credit: UNIVERSITY OF BARCELONA Huntington s disease is caused by a mutation in the Huntingtin gene (HTT), which appears in adults and features motor, cognitive and psychiatric alterations. The origin of this disease has been associated with the anomalous functioning of the mutated protein: mHTT, but recent data showed the involvement of other molecular mechanisms. A new study conducted by the University of Barcelona has identified a type of ribonucleic acid (RNA) as a potential therapeutic target for the treatment of the disease. These are the small RNA, or sRNAs, molecules that do not code proteins but have important functions in the regulation of gene expression. According to the study, sRNAs would take part in the development of the disease, results that shed light on the design of new specific dr

Study identifies potential therapeutic target for treatment of Huntington s disease

Protein alteration contributes to degeneration of neuronal populations in Huntington s disease

Protein alteration contributes to degeneration of neuronal populations in Huntington s disease Protein alteration in the family of lamins causes several diseases, known as laminopathies, such as progeria or precocious aging. A study in which UB researchers have taken part states that alterations in the levels of one of these proteins, lamin B1, contribute to the degeneration of different brain neuronal populations in Huntington s disease. Caused by a mutation in the huntingtin gene, this pathology features involuntary movements, cognitive deficit, and psychiatric disorders, and has no cure yet. According to the study, published in the journal EMBO Molecular Medicine, these results open new therapeutic pathways for the treatment of this disease, since research shows pharmacological normalization of levels of lamin B1 improves the cognitive symptoms in a transgenic model of the disease.

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