Research Fellow Feature: Cameron Niazi

Published on Wed, 02/14/2024

Cameron Niazi
University of Florida, Gainesville, Florida, US

Mr. Cameron Niazi holds a master’s degree in biotechnology from Northwestern University and a bachelor's degree in Biological Science from Cornell University. His journey into DM1 began at Vertex Pharmaceuticals, where he joined the DM1 gene editing group, fueled by his passion for CRISPR/Cas. His research at Northwestern's lab strengthened his expertise in CRISPR/Cas and gene therapy, focusing on targeted epigenetic modifications.

A pivotal six-month stint at AbbVie exposed him to pharmaceutical manufacturing challenges and solidified his decision to avoid a career in process engineering. After two years at Vertex, he pursued a Ph.D. at the University of Florida in Dr. Eric Wang's lab, attracted by the lab's integration of basic science and translational research. Joining the lab provided a personal connection to DM1. Working closely with individuals and families living with DM1 became a profound inspiration for him and the team.

Looking ahead, Mr. Niazi aims to secure a scientist position at a biotech or pharmaceutical company, prioritizing those with DM1 gene editing or gene therapy programs. He indicates that he is dedicated to advancing DM1 gene therapies and intends to stay in the field.

Mr Niazi's project “Leveraging CRISPR/Cas-based Epigenetic Modifications for the Treatment of Myotonic Dystrophy Type 1” represents a modified CRISPR/Cas based “epigenetic silencing” approach that has fewer safety risks compared to most gene editing strategies being pursued for DM1. Epigenetics refers to factors that control how genes are turned on and off. Rather than using “molecular scissors” to go in and cut the problematic DNA. Instead of cutting the problematic DNA, this approach removes CRISPR's "molecular scissors" blades, turning the gene off without physically altering it. The proposed work will be a first step to bringing this promising new technology to the DM1 community and will serve as a valuable test of its overall feasibility as a therapeutic approach.

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