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Search Results - general biology & chemistry

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Novel, long-term treatment of Huntington’s disease by gene therapy
Novel synthetic zinc finger proteins delivered to the brain through the use of viral vectors, efficiently and selectively repress the mutant huntingtin gene in a mouse model of Huntington’s disease long term after a single intervention, and successfully restore the motor coordination to levels comparable to those in healthy controls in an acu...
Published: 08/09/2016   |   Inventor(s): Mark Isalan
Keywords(s): Biotechnology, Gene Therapy, Genetic Diseases, Neurodegenerative Diseases, Neuromuscular & Movement Disorders, Neuroscience, Rare / Orphan Diseases
Category(s): Biotechnology, General Biology & Chemistry, Therapeutics (Biologics & Compounds)
Method to identify new cancer drug targets in spliceosomal components
A new method and tools to map functional interactions of spliceosomal components accurately captures known, and identify new physical and functional associations important for health and disease.The method can be used to infer targets for alternative splicing regulation, and to study the effects on splicing of compounds directed to altered, or dise...
Published: 12/02/2016   |   Inventor(s): Juan Valcarcel, Juan Tejedor, Panagiotis Papasaikas
Keywords(s): Algorithms, Bioinformatics, Genomics, Oncology
Category(s): Methodology, General Biology & Chemistry, Software & Algorithms

 

 

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