Gene and Cell Therapy – Multimedia

Multiple Sclerosis (MS) is a chronic inflammatory demyelinating disorder of the CNS. No successful treatment for MS, but one therapeutic strategy in research is the use of bone marrow-derived mesenchymal stem cells (MSC). We studied a group of MS patients who underwented MSCs, assayed for expression of a transcription factor, FOXP3, as a specific marker of MS amelioration in peripheral blood. qRT-PCR on PBMCs showed higher FoxP3 levels.

Hematopoietic stem cells (HSCs) give rise to all the blood cell types and are important for cell therapy and drug development. During the development of lymphoid and myeloid lineages, HSC differentiate into committed hematopoietic progenitors. Monitoring the expansion and differentiation of HSCs into lineage-commited hematopoietic progenitors is important for research of hematopoiesis and developing therspeutic processes with HSCs

This presentation describes in detail the development and establishment of a current good manufacturing practices (cGMP) facility for the purpose of manufacturing cell and tissue products intended for human use in a hospital setting.

Constitutive TNF-alpha expression is characteristic of the malignant ovarian surface epithelium. Adenoviral mutants hold great promise as gene therapy vectors but their efficacy is hindered by an inflammatory cascade orchestrated by TNF-alpha. We found that delivering TNF-alpha shRNA to ovarian cancer cells using oncolytic adenoviruses could reduce the inflammatory cascade generated by adenoviruses and also had direct anti-tumour activity on the cancer cells.

One major obstacle in stem cell therapy is poor cell engraftment. To address this issue, concurrent investigations were made to develop novel peptides targeting bone marrow and a method to transiently ‘paint’ targeting molecules onto cells. Painting was studied using a model palmitated peptide. Novel bone marrow targeting peptides were sought using in vivo phage display in a locally irradiated mouse model that has an internal control (untreated leg).