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Search Results - neurodegenerative+diseases

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A unique B-cell model system for transdifferentiation and human immune modulation
The cells, derived from a B-cell acute lymphoblastic leukemia cell line, can be easily converted into human macrophage-like cells with 100% efficiency in just few days, recapitulating human macrophage function and biology better and more conveniently than existing model systems. They can be used for basic research and drug development purposes in relation...
Published: 2/19/2019   |   Inventor(s): Thomas Graf, Francesca Rapino
Keywords(s): Autoimmune Diseases, Cardiovascular Diseases, Cell-based Assay, Immune / Inflammatory Diseases, Immuno-oncology, Infectious Diseases, Metabolic Diseases, Neurodegenerative Diseases, Oncology
Category(s): Therapeutics (Biologics & Compounds), Research Tools (cell lines, mice, kits, ABs, ...), Drug Targets & Mechanisms
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 acute...
Published: 2/19/2019   |   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)

 

 

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