Two major breakthroughs in gene editing recently occurred and show how our ability to edit the human genome is rapidly increasing. This episode we dive into how these technologies work and how they were able to treat two diseases. One a rather mundane sounding cholesterol problem and the other an exteremely painful and potentially deadly condition known as sickle cell disease. Both stories invovle a combination of new technologies, mRNA techniques and gene editing either using or inspired by CRISPER. These stories highlight how all of these technologies are combining to open new possibilities, that gene editing may become just a regular treatment as we age, and the less talked about downsides or hidden trade-offs that come with these treatments.

Sources

Base editing, a new form of gene therapy, sharply lowers bad cholesterol in a clincal trial - Science

A Closer Look at the Approval of CRISPR/Cas9 Gene Therapy for Sickle Cell Disease - MedPage Today

New gene therapies confront many sickle cell patients with an impossible choice: a cure or fertility - STAT

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