6th Annual Gene Therapy for CNS Summit – Olga Pietrzyk

Conference & workshop

6th Annual Gene Therapy
for CNS Summit

On Novemver 20-22, 2024 Olga Pietrzyk took part in 6th Annual Gene Therapy for CNS Summit in Boston.

First day she attended the Translational & Clinical Track Workshop. During the workshop, she had the opportunity to learn about selecting the appropriate model for testing AAV vectors, starting from cell culture and extending to mouse models. Two important pieces of information she learned that by choosing the right research model is crucial from the very beginning. For example, a solution that works perfectly in mouse models may not be effective in humans. Therefore, it is worth considering the inclusion of iPSCs in experiments. This is particularly significant for my research, which has primarily focused only on mouse models and the in vivo testing of adeno-associated virus (AAV) vectors.

Conference & workshop

The second thing that Olga found very interesting during the workshop was that there are several parameters that we can test using cell cultures (commercially avaiable cell lines, primary cell culture, iPSCs, organoids and assembloids) such as: AAV tropism, promoter validation, transgene expression testing, and neurotoxicity studies. Additionally, organoids are becoming increasingly popular for AAV vector testing, acting as a bridge between mouse studies and human applications. The brain organoids protocol is being implemented in the PORT project as part of the SAME-NeuroID project, which could represent a new potential application in this field.

Next Olga attended two days of conference. During the two days of the conference, she had the opportunity to attend several excellent talks on the use of AAV vectors in gene therapies for the central nervous system (CNS). Some of the projects are nearing clinical trials, indicating a growing potential for successful gene therapy targeting more neurological diseases.

Conference & workshop

Olga found the information regarding the modification of AAV vector elements, such as capsids and the addition of regulatory elements with promoters, particularly fascinating, as this aligns closely with her own research.

The conference was an intimate event with about 50 attendees (according to my Olga’s calculations). Therefore she had a great opportunity to talk face-to-face with the speakers and other participants. In addition to the regular presentations, there was a roundtable discussion where participants discussed specific issues in small groups. At the end of the discussion, each group shared their main points with the rest of the attendees.

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6th Annual Gene Therapy for CNS Summit – Olga Pietrzyk