Gene and RNA Therapeutic Development

Catalog description

The field of gene and RNA-based therapeutic development has had a profound impact on our ability to treat many unmet medical needs. By targeting the genetic basis of a given disease, it has become possible to regulate, limit, or modify the expression of the associated transcript, and in some cases, completely replace the impacted gene. This course provides a comprehensive exploration into the field of gene-modifying therapeutics, focusing on gene therapy and oligonucleotide therapy. Students will delve into the mechanisms of action, innovative delivery methods, evolving development pipelines, and the strengths and limitations of each therapeutic modality. Students will gain a comprehensive understanding of genetic therapeutics, preparing them to contribute to the advancement of this field and the translation of innovative therapies from bench to bedside. Preference is given to students in the Biotech Master's and Therapeutic Science PhD programs.