Lectures and seminars KI South Distinguished Lecture Series: Aurélie Goyenvalle
Dr. Aurélie Goyenvalle, Director Biotherapy for Neuromuscular Diseases, University of Versailles, INSERM. Her research focuses on developing therapeutic approaches for neuromuscular diseases based on antisense oligonucleotides made of tricyclo-DNA (tcDNA).
Dr. Goyenvalle and her group has taken the leadership in the field of gene therapy and antisense technology to correct the deleterious effects of mutations in various genetic diseases, and in particular for the treatment of neuromuscular diseases such as Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). She has a long-standing expertise in the pre-clinical evaluation of antisense strategies, using both naked antisense oligonucleotides and vectorised approach (AAV-U7). This lecture will describe the therapeutic potential of ASOs made of tricyclo-DNA (tcDNA), which displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration.
Lecture: "Tricyclo-DNA: promising antisense oligonucleo-tides for the treatment of neuromuscular diseases"
Short talk introduction by KI researcher Joel Nordin, MD, Ph.D., Assistant Professor at Karolinska Institutet, the department of Laboratory Medicine: "A Phase I/II study of a Exon 44 skipping drug, in patients with Duchenne muscular dystrophy and novel delivery systems to muscle for gene editing".
The lecture will be followed by an afternoon reception in the Neo entrance hall with possibility to interact and ask questions. A small group will after that be allowed to talk to Dr. Goyenvalle under the leadership of Joel Nordin.
This lecture series will be IRL only. No registration needed.