Low mRNA levels in CHM patients

The article: Association of Messenger RNA Level With Phenotype in Patients With Choroideremia: Potential Implications for Gene Therapy Dose Background The central dogma of biology is simply that DNA makes RNA makes protein. Messenger RNA (mRNA) transfers the information from DNA and is used to create protein. The question this study ultimately seeks to answer is “How much mRNA is needed to have healthy retinal function in CHM patients?” It is important to know this information when conducting gene therapy clinical trials so an effective “dose” can be delivered....

February 23, 2020 · 2 min · Peter Boone