School of Pharmacy
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Maha Muwaffak

, Faculty of Science



Maha Graduated with an MPharm degree (first class honours) from UCL School of Pharmacy. She then completed her pre-registration year working in a community pharmacy, before starting the PhD.

During the master's project she worked under the supervision of Professor Graham Buckton on a project titled" Particle design for inhalation therapy ", which focused on producing engineered lactose carriers that will enhance the aerosolisation performance of micronized drugs. For the first half of the first year of her PhD, she first worked on a mini project titled "Towards cell free therapies: through freeze drying of exosomes". This project aimed to isolate, characterize and freeze dry exosomes, and then evaluate the impact of formulation on the size, number and functionality of the exosomes. She then completed a placement at GSK where she worked on developing a robust GVS based method to quantify low levels of amorphous content in micronized API.

Research Summary

Project title: Cellular factories of enzyme-rich exosomes for neurodegenerative disorders

This project will focus on rare paediatric neurodegenerative metabolic diseases such as Gaucher disease and Batten disease. The outlook for patients suffering such conditions is extremely bleak: for instance, children affected by Gaucher's usually do not reach their 2nd birthday.

In this project, we propose a novel approach to treat these diseases. Cells use a range of mechanisms to communicate, including the production of exosomes. These contain a rich variety of proteins and RNAs within a phospholipid bilayer, and have potent effects in tissue regeneration. They have also been proven to be highly effective drug delivery systems, with the advantages of being non-immunogenic and able to cross the blood-brain barrier.

The precise composition of biomolecules in the exosomes is dependent on the molecules being produced in the cells themselves. In this project, we will exploit this fact to develop a platform for the production of enzyme-rich exosomes, which can then be formulated for the long-term treatment of degenerative disorders.

School of Pharmacy

University of Nottingham
University Park
Nottingham, NG7 2RD

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