Disrupting The Brain Keeper To Allow Silencing Of Deleterious Genes In The Nervous System
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About The Author

Bruno M. D. C. Godinho is a PostDoc at University of Massachusetts Medical School | UMMS · RNA Therapeutics Institute (RTI), specializing in Neurodegenerative Diseases, Nanoparticles, and RNA Interference.

                       

Disrupting The Brain Keeper To Allow Silencing Of Deleterious Genes In The Nervous System

Therapeutic oligonucleotides, such as small interfering RNAs (siRNA), hold great promise for the treatment of genetically-defined disorders, including diseases of the central nervous system (CNS). These oligonucleotide-based drugs effectively and potently modulate gene expression of toxic RNAs and proteins, potentially blocking or reversing disease progression. Despite the significant enthusiasm of the scientific community after their discovery, the premature debut of poorly stabilized siRNA constructs in the clinic resulted in early termination of trials. These events dampened the general interest in...

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