Deciphering disease mechanisms and designing nanomedicine treatments using organoid technology

Targeted therapeutics and nanodevices group  ·  Silvia Muro  /  Pluripotency for organ regeneration group ·  Nuria Montserrat

Research project


The proposed project will encompass a new collaboration between the Targeted Therapeutics and Nanodevices Group directed by Prof. Silvia Muro and the Pluripotency for Organ Regeneration Group led by Dr. Nuria Montserrat, both at IBEC. Focus will be on the use of human pluripotent stem cells and human organoid technologies to study human disease mechanisms and to evaluate the potential of nanomedicine-based applications for their treatment. The use of human organoids is a novel and active field of research, highly relevant in that it offers valuable alternatives to both cellular and animal modeling, with greater promise to impact clinical translation. Nanomedicine also encompasses state-of-the-art concepts and methods to design nanotechnology-based therapies aimed to increase the precision, efficacy, and safety of treatment. By comprising both aspects, the project offers a unique opportunity to impact both fundamental and translational sciences while training the student in an ample variety of techniques and disciplines.

A relevant example to illustrate the use of these technologies is the case of kidney pathology caused by inherited maladies, such as Fabry disease, a lysosomal storage disorder due to genetic efficiency of alpha-galactosidase enzyme. Although mouse models exists which represent a major tool for understanding the mechanisms responsible for this human syndrome, differences between these species represent a major obstacle to translate promising findings from the mouse into the human setting. Human pluripotent stem cells (hPSCs) offer a unique opportunity to overcome this problem since they can be used to model human disease and to evaluate the practical potential of novel therapies aimed at treatment. In the last years the Montserrat lab has deciphered how to generate different kidney cell populations from hPSCs and how to derive from them organoids, to investigate the molecular mechanisms leading to disease gestation in order to develop approaches targeting those processes. In turn, the Muro lab has developed diverse nanotechnologies aimed to target lysosomal storage disorders, including Fabry and other diseases, to enable efficient replacement of genetically deficient enzymes by functional recombinant ones, providing an avenue for treatment. Both efforts will be united in this project to provide novel avenues to study disease development and treatment efficacy.

 

Job position


We are looking for highly motivated PhD students to work on the fundamental understanding of the pathological alterations in the kidney occurring as a consequence of genetic lysosomal storage disorders (i.e., Fabry disease) and to develop novel nanomedicine solutions for treatment of said conditions. It is expected that the student will also contribute to common lab supporting tasks, research collaborations, recording and reporting processes, as well as publications and other scientific communications derived from this work.

The student will use a wide range of concepts and methods encompassing the fields of biochemistry, molecular and cellular biology, embryological and molecular biology techniques, analytical tools, drug targeting, nanotechnology, etc.

Applicants should possess a strong research background in molecular and cell biology, biotechnology, or associated disciplines. Specific experience in the fields of stem cells development/biology and/or nanoengineering will be an advantage.