Dr. Manouchehr Amanat is currently working as post-doctoral research fellow at Kennedy Krieger Institute and Johns Hopkins Medical Institute. He achieved his Medical Doctorate degree from Tehran University of Medical Sciences in Tehran, Iran. He is developing novel RNA therapeutic agents such as antisense oligonucleotides in neurodevelopmental disorders. He is skilled at cell culture and growing induced pluripotent stem cells, advanced statistical methods and machine learning models using Python, and designing clinical trials.
Project: Antisense Oligonucleotide Therapy in X-linked Adrenoleukodystrophy
This project focuses on the application of antisense oligonucleotide therapy in the context of X-linked adrenoleukodystrophy (X-ALD). The primary objective of this research is to elevate the expression of the ABCD2-related protein as a compensatory mechanism for the deficiency of functional ABCD1-related protein in X-ALD patients. To achieve this, we have designed specific ASOs at Kennedy Krieger Institute to directly target ABCD2 mRNA to enhance protein expression. The experimental approach involves culturing fibroblasts derived from X-ALD patients, introducing ASOs, and subsequently quantifying the levels of ABCD2 mRNA and very long-chain fatty acids. A similar methodology will be applied to microglia cells derived from induced pluripotent stem cells. Ultimately, this project represents a significant step towards addressing the challenges posed by X-ALD.