The real power of gene editing is being able to choose exactly where to edit a genetic disorder, as easily as correcting a tiny typo in a text.

With its 25 years of expertise, Cellectis was the first company to translate gene editing tools into potentially life-saving therapies. They invented the allogeneic approach (which is the transfer of cells from one individual to another). Cellectis’ technology, TALEN®, can make very precise edits, with limited to no off-target effects and we are learning more about it in this episode.

To do so, we are joined by Dr. Julien Valton, Vice President Gene Therapy at Cellectis.

Overview of Cellectis:

• Celebrating 25 years of innovation in gene editing.
• Overview of Cellectis’ journey and contributions to the field.

Understanding TALEN Technology:

• Explanation of what TALEN stands for and how it works.
• Comparison of TALEN with other gene editing technologies like CRISPR.
• Advantages and disadvantages of using TALEN over CRISPR.
• TALEN's intellectual property status and whether other companies are working on this technology.

Chimeric Antigen Receptor (CAR) T-cells:

• Introduction to CAR T-cells and their role in gene editing with TALEN technology.

Recent Research and Innovations:

• Discussion on the new paper co-authored by Dr. Valton on using TALEN technology to edit hematopoietic stem and progenitor cells.
• Explanation of the “promoterless intronic gene editing approach” and how it differs from usual methods.
• Reasons for choosing Mucopolysaccharidosis MPS type I for the study.
• Insights into the decision to trigger gene expression only after cells have turned into the myeloid lineage.
• Remarkable findings of “corrected cells” in the brains of mice and the implications for treating metabolic and neurological disorders.

Implications and Future Directions:

• Potential therapeutic applications in the treatment of metabolic and neurological diseases.
• Discussion on the recent partnership agreement with AstraZeneca.
• Specific diseases targeted with TALEN in the near future and the goals of the collaboration with AstraZeneca.

Conclusion:

• Final thoughts from Dr. Valton on the future of gene editing and Cellectis’ role in advancing this field.

Stay tuned for the next new episode of DNA Today next Friday! New episodes are released every Friday. In the meantime, you can binge over 290 other episodes on Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “DNA Today”. Episodes since 2021 are also recorded with video which you can watch on our YouTube channel, this includes some episodes recorded at NBC Universal Stamford Studios. 

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