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White Paper

The Vehicle of Success - An Adaptive Approach to Genetic Medicines

Introduction

There are various approaches to gene therapy, including inactivating a malfunctioning mutated gene, replacing a disease-causing mutated gene with a healthy copy, or introducing a new gene to help fight a disease. “There are numerous mechanisms of gene therapy, including gene silencing with RNA interference, mRNA-based therapies that lead to increased protein expression, and CRISPR-Cas9 gene-editing tools which can correct for mutations that may be causing a disease,” said Dr. Amiji.

Dr. Mansoor Amiji, University Distinguished Professor, Professor of Pharmaceutical Sciences and Professor of Chemical Engineering at Northeastern University, Boston, MA, USA recently discussed the various approaches to gene therapy, including viral and non-viral vector delivery, and how regulatory agencies are ensuring these products are safe and effective for human use.