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Jennifer Erwin is a molecular geneticist and neuroscientist who studies how environmental and genetic variations affect the human brain in health and disease. Her research group uses a hybrid of human stem cell models, post-mortem tissue and computational approaches to interrogate the contribution of epigenetic and somatic mosaicism to brain development. Jeannie T. Lee at Harvard.

Using AAV vectors, his group has been developing gene therapies for neurodegenerative diseases. His expertise has contributed to clinical trials of gene therapy for AADC deficiency. Her clinical interests have focused on the immune responses in children with hematologic disorders and immune dysfunction in patients with sickle cell disease. In addition to her deep clinical expertise, Dr. Wong has a proven track record of drug development spanning clinical research to global medical affairs activities to support approved and pipeline therapeutic agents.

After a year career in virology involving the molecular characterisation of positive-strand RNA viruses, he changed his research focus to the genetic modification of haematopoietic and mesenchymal stem cells using adenoviral, lentiviral and hybrid vector technology. This led him to epigenetics, where his work on mobile genetic elements and their controllers is providing new insights into human biology and promising leads for precision medicine. He has a long standing research and clinical interest in development and application of gene therapy for patients with primary immunodeficiency, especially Wiskott-Aldrich Syndrome WAS , disorders of innate immunity, and Autoimmune Lymphoproliferative Syndrome.

She has been working on academically sponsored Clinical Trials for 18 years. The portfolio includes cell and gene therapies, including CAR T-cell therapies. She manages gene therapy projects at different stages of development ranging from preclinical, to clinical, to registration and market access. Winner of edition of TopRA Award in the Future category, Michela offers up to date knowledge of the most recent regulation in ATMPs and is frequently invited as speaker at international events on the subject.

Novel Technologies for Clinical Applications

His laboratory is focused on in vivo gene therapy. He is a named inventor on several patents in the field and has significant experience in GxP assay development, routine testing, manufacturing, manufacturing development and analytical development. As CTO, James has overall leadership responsibility for both Manufacturing responsible for routine GMP production, supply chain and oversight of technical programmes and Manufacturing Development responsible for manufacturing process development, technical support, new process introduction and technology.

In , she led an international research team to develop a needle-free adenovirus based Ebola vaccine, patenting a novel method for stabilising the vaccine, reporting the first long-lasting protection with a single dose of the vaccine in non-human primates. His research focuses on how the chronically damaged liver regenerates and how these mechanisms become deranged in liver and bile duct cancer. Stuart is a consultant Hepatologist at the Scottish Liver Transplant Unit and has clinical interests in liver failure, cancer and liver transplantation.

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Ron Philip has over 20 years of experience in the pharmaceutical industry in multiple therapeutic categories and global markets at Spark Therapeutics, Pfizer, Wyeth and Merck. Ron Philip is currently Senior Vice President, Head of Global Commercial at Spark Therapeutics and responsible for leading commercial operations which includes sales, marketing, diagnostics, market access and patient services functions for inline products and managing commercial development activities for late stage pipeline assets.

His has a long-standing interest in adenovirus vectorology and more particularly in the manipulation of adenovirus capsid to control the tropism and toxicities of adenovirus-derived vectors. His laboratory based at Institut Gustave Roussy Villejuif , France is currently focusing on i a vaccination strategy based on epitope display on adenovirus capsid and ii the combination of oncolytic adenoviruses with chemotherapeutics. Since graduating in pharmaceutical chemistry from the University of Padua, Paola has now worked in Quality Assurance for more than 25 years.

Martina is responsible for advising companies on drug development issues, assessing clinical trials and reviewing marketing authorisations, and contributes to the development of guidelines in the ATMP field. Ian M. MacDonald is a product of the public school system of Canada. His area of clinical and research interest is inherited retinal disorders, in particular, disorders of the macula and choroideremia.

He has conducted studies in the field of cell therapy in neuroscience. Since , he is also vice-president of the Swiss Institute for Cell Therapy. Auricchio is a member of multiple editorial, the recipient of several prestigious scientific and has held ERC grants since More recently, his lab has also developed a very active program researching the role of exosomes in cancer progression.

Matthias did his Ph. She developed a dual experience in discovery biology in both academia and industry, and published in top-ranked peer-reviewed scientific journals. Fessart is an expert in 3D model of stem-cell-derived organoids, tumor niche and secretome. Since his PhD at the University of Naples, Federico II and a postdoc at Baylor College of Medicine, Vince has raised more than 5 million euros for research, produced tens of scientific papers and patents and has co-founded a multimillion spin-off company together with the University of Helsinki VALO therapeutics.

He received his Ph.


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His research interests are at the interface of viruses and the human immune system, with a focus on molecular engineering of desired viral and immune functions. He is currently exploring innovative technologies to harness innate immunity for therapeutics, including AAV gene therapy and mRNA vaccines. He holds a degree in Veterinary medicine and a PhD in biochemistry and molecular biology from the Autonomous University of Barcelona. He has expertise in several viral vector platforms for gene therapy, such as adenoviral vectors, helper-dependent adenoviral vectors and adeno-associated vectors and he has made significant contributions to field of in vivo gene transfer in small and large animal models of diseases.

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He has a broad background in stem cell biology and hematology, with expertise in in vivo approaches, molecular and cellular biology, and immunology. Goals of his work are to translate discoveries in basic research into practical medical applications. The main research focus of her lab is the development of cell and gene therapy approaches to treat cancer. She pioneered the clinical application of suicide gene therapy applied to allogeneic stem cell transplantation and more recently developed innovative approaches to redirect T cell specificity against cancer with genetic manipulation and genome editing.

1. Introduction

She is the recipient of several prestigious scientific awards. James Thomson Additionally, she has served as a Scientific Advisor to multiple boards. Dr Verhoeyen is an expert in development of novel lentiviral vector pseudotypes for the transduction of human T cells, B cells and hematopoietic stem cells for gene therapy purposes as also the engineering of novel vectors for in vivo transduction of hematopoietic cells.

Biomaterials for Tissue Engineering

Previous Next Close. Not a member? View on Facebook. Next the team performed a series of culture-based experiments to unveil the precise molecular mechanism responsible for the regenerative effects of the laser treatment.


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  • It turns out that a ubiquitous regulatory cell protein called transforming growth factor beta-1 TGF-1 played a pivotal role in triggering the dental stem cells to grow into dentin. TGF-1 exists in latent form until activated by any number of molecules. Here is the chemical domino effect the team confirmed: In a dose-dependent manner, the laser first induced reactive oxygen species ROS , which are chemically active molecules containing oxygen that play an important role in cellular function.

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    Nailing down the mechanism was key because it places on firm scientific footing the decades-old pile of anecdotes about low-level light therapy LLLT , also known as Photobiomodulation PBM. Since the dawn of medical laser use in the late s, doctors have been accumulating anecdotal evidence that low-level light therapy can stimulate all kind of biological processes including rejuvenating skin and stimulating hair growth, among others. The clinical effects of low-power lasers have been subtle and largely inconsistent.

    The new work marks the first time that scientists have gotten to the nub of how low-level laser treatments work on a molecular level, and lays the foundation for controlled treatment protocols. Next Arany aims to take this work to human clinical trials. Noninvasive laser therapy could radically shift dental treatment and lead to a host of broader clinical applications in regenerative medicine A Harvard-led team is the first to demonstrate the ability to use low-power light to trigger stem cells inside the body to regenerate tissue, an advance they reported in Science Translational Medicine.

    These tooth models show the relative size comparison of a human tooth versus a rat tooth — to give an idea of the technical challenges involved with performing dentistry on such a small scale.