Gene and Cell Therapy – Multimedia

Joseph Wu, Director, Stanford Cardiovascular Institute, speaking at Stem Cells in Drug Discovery.

The functional network of human induced pluripotent stem cell (hiPSC)-derived neurons is a potentially powerful in vitro model for evaluating disease mechanisms and drug responses. However, the culture time required for the full functional maturation of individual neurons and networks is uncertain. We investigated the development of spontaneous electrophysiological activity and pharmacological responses for over 1 year in culture using multi-electrode arrays (MEAs).

Raj Batra, University of California Los Angeles, speaking at Diagnostics & Biomarkers 2015.

The research reviews current knowledge of the genetic causes of Leber's congenital amaurosis (LCA) and the development of a gene therapy intervention for LCA. The efficacy as well as the limitations of this form of treatment are evaluated in detail and recommendations are made for the future directions of gene therapy for Leber's congenital amaurosis.

Study objective was to develop methodologies for gram scale synthesis of messenger RNA (mRNA) for gene therapy applications, as well as scalable purification methods that yield highly expressed, persistent and non-toxic mRNA.

This study looks at off-target cleavage of Zinc Finger Nucleases (ZNFs) in Drosophila in an attempt to analyze potential cleavage spots, with a view to designing more efficient ZFNs.

Regulatory authorities are active to ensure a good regulatory environment for somatic cell therapy-, gene therapy- and tissue engineering products (in the EU known as ‘Advanced Therapy Medicinal Products’). Both the EMEA and the FDA have, amongst others, issued guidelines to address the specific aspects of these products

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.