NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
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Written by Carrie A. Sheeler Edited by Dr. Ronald A.M. Buijsen RNAi reduces levels of disease-causing Ataxin-1 in SCA1 model mice, easing symptoms of disease when injected both before and after symptom onset. Lowering the amount of the disease-causing mutant Ataxin-1 protein in affected cells and tissues improves symptoms of Read More…
A common nuisance for bacteria is the bacteriophage: a virus that uses the internal machinery of a bacteria to replicate its own genetic material. Bacteriophages do this by latching onto bacteria and injecting their DNA into the cell. As the cell grows and divides, the bacteriophage’s hope is that their Read More…
Gene therapy is using nucleic acids to treat a genetic disorder. These nucleic acids can be designed in a variety of ways to achieve the same therapeutic outcome. Gene therapy tools can be used to correct a mutant gene by one of three ways: Expressing a healthy copy of a Read More…
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