Gene regulatory mechanisms of neocortex development and evolution & CLEANSIGHT: from basic human RPE cell biology to disease modeling and therapy discovery

Date

Apr 5, 2019

Time

4:00 PM - 5:00 PM

Speaker

Dr. Mareike Albert

Affiliation

Dr. Seba Almedawar

Language

en

Main Topic

Biologie

Other Topics

Biologie, Medizin

Description

Speaker: Dr. Mareike Albert Title: Gene regulatory mechanisms of neocortex development and evolution AbstractEpigenetic mechanisms play a pivotal role in orchestrating the behavior of stem cells during development. We are interested in the mechanisms that regulate gene expression in neural stem and progenitor cells, which have implications for our understanding of neocortex development and evolution as well as neural stem cell regulation. Speaker: Dr. Seba Almedawar Title: CLEANSIGHT: from basic human RPE cell biology to disease modeling and therapy discovery AbstractInherited and age acquired retinal degenerative diseases (RD) are the primary cause of vision loss in the industrialized world. The retinal pigment epithelium (RPE) is implicated in many RD, including age related macular degeneration (AMD), Best1 disease and retinitis pigmentosa. A decade ago, studying those diseases in vitro was challenging due to limited access to patient RPE, which hampered the development of therapy. In CLEANSIGHT, we use human pluripotent stem cells derived RPE to study and model RD and develop therapeutic approaches using small molecules or cell replacement therapy. Our established screening platform revealed stimulators of photoreceptor outer segment phagocytosis, which is a major disease causing phenotype in RD. Currently we are validating our favorite hit in vivo and transferring our finding to industry for drug development. In parallel, we have made the first steps to transfer our protocol to more defined and animal origin free media, which is required for cell replacement therapy. Additionally, in collaboration with Prof. Marius Ader and Miltenyi Biotec, we have identified cell surface markers that can be used to purify RPE cells from remnant stem cells before transplantation, which is a challenge in the field. By establishing both small molecules and cell replacement therapy approaches, the best treatment strategy can be chosen based on the RD stage (early vs late), on a patient-to-patient basis.

Links

• to announcement of the organizer

Last modified: Apr 5, 2019, 2:08:30 AM