POLR3-Related Leukodystrophy: Exploring Potential Therapeutic Approaches.
Finding a cure or targeted therapy for 4H Leukodystrophy is a priority for families and researchers. Unfortunately, as demonstrated , we have limited knowledge of the exact underlying mechanisms leading to 4H Leukodystrophy, making it difficult to know how best to treat it. This 2020 academic article explores stem cell transplantation, gene therapy, and gene editing as potential future approaches. If you’re having a hard time with this longer paper or simply aren’t interested in all the scientific details, you might consider looking at the two figures and skipping to the conclusion. It offers a nice overview, looking ahead. There is also a list of abbreviations at the end if you get stuck on one.
Perrier, Stefanie et al. “POLR3-Related Leukodystrophy: Exploring Potential Therapeutic Approaches.” Frontiers in cellular neuroscience vol. 14 631802. 28 Jan. 2021, doi:10.3389/fncel.2020.631802