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Gene and Cell Therapy – Multimedia

Pharmacological Responses in Cultured Human iPSC-Derived Cortical Neurons Using Multi-Electrode Array   content piece image
Poster

Pharmacological Responses in Cultured Human iPSC-Derived Cortical Neurons Using Multi-Electrode Array

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).
Video

A Novel Vertically-Integrated Platform for the Discovery and Molecular Characterization of Lung Cancer Stem Cells

Raj Batra, University of California Los Angeles, speaking at Diagnostics & Biomarkers 2015.
Efficacy of Gene Therapy in Restoring Vision in Leber's Congenital Amaurosis content piece image
Poster

Efficacy of Gene Therapy in Restoring Vision in Leber's Congenital Amaurosis

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.
Gene Expression from Pseudourine and 5-Methylcytidine Modified Messenger RNA content piece image
Poster

Gene Expression from Pseudourine and 5-Methylcytidine Modified Messenger RNA

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.
Defining off-target cleavage in a pair of Zinc Finger Nucleases content piece image
Poster

Defining off-target cleavage in a pair of Zinc Finger Nucleases

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 requirements in the development of advanced therapy products (cell-, gene therapy, tissue engineering products) content piece image
Poster

Regulatory requirements in the development of advanced therapy products (cell-, gene therapy, tissue engineering products)

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
FOXP3 Gene Expression in Multiple Sclerosis patients before and after Mesenchymal Stem Cell therapy content piece image
Poster

FOXP3 Gene Expression in Multiple Sclerosis patients before and after Mesenchymal Stem Cell therapy

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.
High-Throughput Multiplexed Assay for Analysis of Hematopoietic Stem Cells Differentiation and Hematopoietic Toxicity content piece image
Poster

High-Throughput Multiplexed Assay for Analysis of Hematopoietic Stem Cells Differentiation and Hematopoietic Toxicity

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
Cell Therapy Product Manufacturing in a Hospital Setting content piece image
Poster

Cell Therapy Product Manufacturing in a Hospital Setting

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.
Targeting Inflammatory Cytokines Using Adenoviruses: gene delivery of biological therapies in ovarian cancer content piece image
Poster

Targeting Inflammatory Cytokines Using Adenoviruses: gene delivery of biological therapies in ovarian cancer

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.
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