Workshop 2. NET-RARE: Humanized Models for Studying Rare Diseases
Friday, July 4 @ 9:30 am–1:30 pm
In this second workshop, we will explore how humanized and bioengineered models can replace 2D cultures and murine models to better understand and replicate rare disorders. The goal is to examine the latest approaches for studying rare diseases in personalized human models. The opening seminar will feature Juanma Fernández from IBEC’s “Biosensors for Bioengineering” group. He will present a 3D model designed to study various muscular diseases, along with Organ-on-Chip (OOC) technology and a biosensor platform for detecting biomarkers of the pathology. Next, Maria del Mar Mañú from VHIR’s “Childhood Cancer and Blood Disorders” group will introduce their new personalized model for studying rare anaemia disorders. Her team, in collaboration with IBEC, has developed a spleen-like microfluidic filtering unit to model disease progression and create a diagnostic device for prognosis and patient stratification. Following this, international speaker Francesco Saverio Tedesco, leader of the Stem Cells and Neuromuscular Regeneration Laboratory at University College London, will present his research on enhancing muscle regeneration to develop treatments for severe and incurable childhood diseases, like muscular dystrophies. His team has developed a novel human engineered muscle model for advanced in vitro modelling of muscle disorders involving extracellular matrix, including COL6-related dystrophies. After a networking break, the next talk will be given by Laura Siles from the Instituto de Microcirugía Ocular (IMO) in Barcelona. The group develops 3D retina models using induced pluripotent stem (iPS) cells and has extensive expertise in gene editing with CRISPR technology applied to patient-derived iPS cells. Finally, José Antonio del Rio and Pol Picón Pagès from IBEC’s “Molecular and Cellular Neurobiology” group will share their research on neurodegenerative diseases.
Scheduled Program:
9.30-9.40: Welcome and short introduction of the workshop
9.40-10.10: Juanma Fernández-Costa, IBEC Group “Biosensors for Bioengineering”
10.10-10.40: National invited speaker: María del Mar Mañú Pereira/Anna Collado Gimbert (tbc) from the VHIR group “Childhood Cancer and Blood disorders”.
10.40-11.30: International invited speaker: Francesco Saverio Tedesco, professor of Neuromuscular Biology and Regenerative Medicine, Department of Cell and Developmental Biology, University College London (UK).
11.30-12.00: Coffee Break and Networking
12.00-12.30: National invited speaker: Laura Siles from the Instituto de Microcirugía Ocular (IMO)
12.30-13.00: José Antonio del Rio/Pol Picón Pagès, IBEC group “Molecular and Cellular Neurobiology”
13.00-13.10: Remarks and Closing
