Scientific Program

Conference Series Ltd invites all the participants across the globe to attend 7th International Conference and Exhibition on Cell and Gene Therapy London,UK.

Day 1 :

  • Cell Therapy | Gene and Cell Therapy for Rare & Common Diseases | Stem Cell Therapies | Ethical Issues in Cell and Gene Therapy | Gene editing technology | Tissue Science & Regenerative Medicine | Clinical Trials on Cell & Gene Therapy | Advanced Gene Therapeutics | Cell Culture and Bioprocessing

Session Introduction

Shuying Yang

University of Pennsylvania, USA

Title: Stem Cell Therapy: Bone Regeneration for Critical Size Bone Defects
Speaker
Biography:

Dr. Yang is an Associate Professor in Department of Anatomy & Cell Biology, UPenn Dental School. She received her MD and PhD in China, and then completed postdoctoral fellowships in bone biology at University of Michigan, and later The Forsyth Institute &Harvard School of Dental Medicine. She joined Department of Oral Biology in SUNY at Buffalo in 2008 as an assistant professor and then became a tenured associate professor in 2014. She moved to Penn in 2016. She is an author of over 50 papers published in international journals including Nature Communication, Cell Death & Differentiation, JBMR, Human Molecular Genetics, a reviewer for numerous journals and NIH and other study sections, and editorial board of several journals. Dr. Yang’s lab has pursued innovative research on the mechanisms of bone formation and resorption, including craniofacial and bone development, metabolism and mechanostransduction, and stem cell-mediated bone regeneration.

Abstract:

Critical-sized bone defects (CSBDs) are wounds that cannot be spontaneously bridged and result in the formation of fibrous connective tissue rather than bone when left untreated. Clinical therapies of CSBDs represent a great challenge for orthopedic and craniomaxillofacial surgeons, because current treatments rely on grafting materials such as autografts, allografts, or xenografts. However, recent advancements in stem cell-based bone repair and regeneration have shown great promise in animal models and clinical studies. In this talk, the speaker will report her lab findings including using new injectable and moldable nano calcium sulfate scaffolds (nCS), and growth factor conjugated controllably released fibrin gel system to deliver mesenchymal stem cells, endothelial progenitor cells, genes, growth factors and PRP for promoting angiogenesis and bone regeneration in critical-sized bone defect models.

Speaker
Biography:

Dr Paul von Hoegen is Senior Director Business Development at CTL Europe. Before he was leading the European science based business development for JPT Peptide Technologies.  He managed biotechnology and biobanking projects at The Weinberg Group in Brussels (Belgium) and served as scientific leader in several biotechnology companies (Biovector Therapeutics, Pharmexa and Europroteome) working in immunology and vaccine development for infectious disease and cancer. Dr. von Hoegen studied Biology at the University Cologne, Germany, and obtained his PhD in Immunology at the University of Heidelberg, Germany. He worked at the Stanford University and SyStemix, USA and headed a junior group in cancer vaccination at the German Cancer Centre Heidelberg, Germany. In 1995 he joined GSK Biologiclas where he headed the cellular immunology group and initiated several therapeutic vaccine projects. Dr. von Hoegen is author and co-author of numerous publications and been involved in multiple R&D collaboration projects.

Abstract:

Cancer and infectious diseases can be treated by enhancing immune surveillance.  Suitable immune therapies can rely on in vitro expansion of the reactive lymphocytes through cell line development, e.g., the expansion and activation of natural killer cells, or of antigen-specific T cells for T cell therapy.  Gene therapy is suited for the induction or enhancement of immunity to cancer and infectious agents via vaccination, or the generation of chimeric antigen receptor (CAR)-T cells.  All these immune therapeutic measures have in common the induction and expansion of rare effector cell types, along with gearing these cells towards the desired effector functions. Therefore, measurements of frequencies and functions of the specific effector cells are key for judging therapeutic potentials.  During my talk I will focus on a technology that permits to do that by multiplex ImmonoSpot® measurements establishing the cytokine signatures and cytolytic potential of antigen-specific T cells and NK cells.  This technology has been validated for immune monitoring in clinical trials and also lends itself for batch testing of cells to be injected for cell therapy.    

Speaker
Biography:

 

I have been at Wake Forest University for 30 years. During that time, my research focus has been women’s health. While now a faculty member of Regenerative Medicine, my past research focused on the use of animal models to explore the effects of aging and sex hormones on the cardiovascular and urogenital systems.  When moving to the Wake Forest Institute for Regenerative Medicine (WFIRM), my focus remained on women’s health and was funded for 4 years and now funded for an additional 4 years to explore the utility of cell therapy for urinary incontinence – using nonhuman primates as the animal model.

Abstract:

It is not unusual for animal models of disease to inaccurately predict clinical outcome of clinical studies.  One such example is stem cell therapy for stress urinary incontinence (SUI) where preclinical studies report almost complete remission of symptoms, whereas clinical studies report only around 50% remission in 50% of patients.  The answer is most likely explained because animal models (which create acute SUI in relatively young animals) do not represent the most common clinical scenario where SUI is most common as a chronic disease in peri/post-menopausal women with co-existing risk factors such as obesity and type-2 diabetes. To better predict the effects of cell therapy for UI, we developed a cynomolgus monkey model of urinary incontinence (surgical nerve and muscle damage to the urinary sphincter complex) that reproduces the functional and structural changes in the urinary sphincter complex seen in women with clinical SUI. In these studies, we modeled both acute and chronic SUI in younger and older female NHPs with varying degrees of estrogen deficiencies and impaired glucose/insulin metabolism.  With an n=6/experimental group, autologous skeletal muscle precursor cells (skMPCs) were isolated from a muscle biopsy, expanded to 5 million cells and injected directly into the urinary sphincter complex of NHPs with SUI. skMPCs almost completely restored sphincter muscle content and urethral pressures in younger (5-8years) NHPs (p<0.05 vs SUI/no treatment), but not older (15-28 years) NHPs (p>0.05 vs. SUI). This same pattern of efficacy was observed in NHPs with acute vs. chronic SUI, in intact vs. ovariectomized NHPs; in normal cycling dominant NHPs vs. dysmenorrheic subordinate NHPs and in normal weight/normal glucose metabolism vs. heavier impaired glucose/insulin ratio NHPs. Thus, there are multiple determinants of cell therapy efficacy that can be modeled in NHPs and are critical to translational applicability of regenerative medicine approaches to tissue repair.

Speaker
Biography:

Kathleen Hefferon received her PhD from the Department of Medical Biophysics, University of Toronto.  She is currently working  as a postdoc at the Department of Food Sciences at Cornell University. Kathleen is also a visiting faculty member at the University of Toronto.  Kathleen has four patents and has edited six books. She has written two books on plants and human health, and has recently been commissioned to work on a second edition for one of them. Kathleen is currently the co-editor of the Encyclopedia of Food Security and Sustainability. Kathleen has been selected for the Fulbright Canada Research Chair in Food Security at the University of Guelph beginning in January, 2018.  Her research interests include agricultural biotechnology, global public health and food/energy security.

Abstract:

For over two decades now, plants have been explored for their potential to act as production platforms for biopharmaceuticals, such as vaccines and monoclonal antibodies. Without a doubt, the development of plant viruses as expression vectors for pharmaceutical production have played an integral role in the emergence of plants as inexpensive and facile systems for the generation of therapeutic proteins. More recently, plant viruses have been designed as non-toxic nanoparticles which can target a variety of cancers and thus empower the immune system to slow or even reverse tumor progression. The following presentation describes the employment of plant virus expression vectors for the treatment of some of the most challenging diseases known today. The presentation concludes with a projection of the multiple avenues by which virus nanoparticles could impact developing countries.

Speaker
Biography:

To be updated soon

Abstract:

The overall objective of the present research was to develop a nanocarrier system for non-invasive delivery to brain of molecules useful for gene therapy.  Manganese-containing nanoparticles (mNPs) carrying anti-eGFP siRNA were tested in cell cultures of eGFP-expressing cell line of mouse fibroblasts (NIH3T3). The optimal mNPs were then tested in vivo in mice. Following intranasal instillation, mNPs were visualized by 7T MRI throughout brain at 24 and 48 hrs.  mNPs were effective in significantly reducing GFP mRNA expression in Tg GFP+ mice in olfactory bulb, striatum, hippocampus and cortex. Intranasal instillation of mNPS loaded with dsDNA encoding RFP also resulted in expression of the RFP in multiple brain regions.  In conclusion, mNPs carrying siRNA, or dsDNA  were capable of delivering the payload from nose to brain. This approach for delivery of gene therapies to humans, if successful, will have a significant impact on disease-modifying therapeutics of neurodegenerative diseases.

Yuan-Chuan Chen

National Applied Research Laboratories, Taiwan

Title: Ethical Issues in Cell and Gene Therapy using CRISPR/Cas9 system
Speaker
Biography:

Yuan-Chuan Chen completed his PhD in Biochemistry at the University of California, Berkeley, USA in 2015/05 and had postdoctoral studies at the Taiwan Food and Drug Administration (TFDA) from 2015/07 to 2017/05. He has published 12 co-authored articles in peer-reviewed journals and 3 chapters in 3 reputed books including the fields of basic science, biomedicine, and related policy/regulation. He is now an assistant researcher at National Applied Research Laboratories (NARL), Taiwan. His research is focusing on the perspectives and challenges of cell and gene therapy based on gene editing technologies such as the CRISPR/Cas9 system.

Abstract:

The CRISPR/Cas9 system has revived many safety issues with our living system and all organisms such as environmental polution, ecological calamity, risk assessment and genome editing in germline. The new concern is the simplicity, rapidity, accuracy and economics of CRISPR/Cas9  for cell and gene therapies, with the possibility of ethical issues. These issues may include balance of benefits and risks, compatibility of private interests and the public good, random manipulation of genes, and commercialization of human therapy. CRISPR/Cas9 has multiple advantageous applications, but hazards are unavoidable. A scientific evaluation system is needed to assure that benefits are greater than risks. There have been many disputes and frictions among companies over patenting CRISPR/Cas9 for human therapy because many commericial interests are involved. An agreement to regulate patent holders and licensees to consider the public good is required. Altering a gene may produce new or undesired species, and lead to  unknown or unpredictable diseases. The use of CRISPR/Cas9 in gene editing should be deliberately evaluated and strictly controlled, especially in human germline. CRISPR/Cas9  has been demonstrated promising for many diseases treatment; however, cell and gene therapies usually require a long course of treatment and cost much. The therapy should be affordable for all patients to avoid being privileged or prioritized for some people. Ethics is not a barrier to science but to allow science to develop long term and perfectly. It is necessary to have a  public communication over the social, legal and ethical implications with the policy/regulatory needs of the system.

Speaker
Biography:

 A Yilmazer is working as an Assistant Professor in the Biomedical Engineering Department of Ankara University. She has been the Vice Director of the Stem Cell Institute of Ankara University since December 2016. She completed her MSc degree in Cancer Immunotherapy from the University of Nottingham (UK) and obtained her PhD in the Nanomedicine Lab based in the School of Pharmacy, University College London. She has published papers on nanomedicine, cancer therapy and cellular reprogramming in various distinguished journals

Abstract:

Graphene, a two-dimensional, single-layer sheet of sp2 hybridized carbon atoms, carbon-based material with physical properties such as electronic conductivity, thermal stability, mechanical strength. There are different forms of grapheme including graphene oxide (GO). Different forms can be easily processed and functionalized. These functionalized forms can have different interactions with biological structures. Graphene based materials have different applications in the biomedical field, one of which is cancer therapy. This talk will summarize various studies involving graphene based materials in cancer research. Specific focus will be placed on photocatalytic and photodynamic therapies involving graphene derivatives. Furthermore, the importance of using 2-dimensional or 3-dimensional cultures in these graphene based cancer therapy protocols will be discussed in detail.

Speaker
Biography:

Dr Cherie Ann Kruger completed her docterate two years ago and is currently employed at the University of Johannesburg, Laser Research Centre, Faculty of Health Sciences as a Researcher. Her research interest lies within the scientific field of Photodynamic Therapy), whereby she investigates the uses of various photosynthetic chemotherapeutic agents to target and kill cancer cells. Currently, she is investigating the development of multicomponent nanoparticle based systems as efficient, specific and targeted drug delivery systems for the photodynamic treatment of various cancers. She has successfully published 10 peer reviewed scientific journal, with 547 citations and H-index of 3.

Abstract:

Photodynamic therapy (PDT) involves interaction of a photosensitizer, light, and molecular oxygen which produces singlet oxygen and subsequent tumour eradication. The development of second generation photosensitizers, such as phthalocyanines, has improved this technology. Customary monolayer cell culture techniques are, unfortunately, too simple to replicate treatment effects in vivo. Multicellular tumour spheroids may provide a better alternative since they mimic aspects of the human tumour environment. This study aimed to profile 84 genes involved in apoptosis following treatment with PDT on lung cancer cells (A549) grown in a monolayer versus three-dimensional multicellular tumour spheroids (250 and 500 µm). Gene expression profiling was performed 24 h post irradiation (680 nm; 5 J/cm2) with zinc sulfophthalocyanine (ZnPcSmix) to determine the genes involved in apoptotic cell death. In the monolayer cells, eight pro-apoptotic genes were upregulated, and two were downregulated. In the multicellular tumour spheroids (250 µm) there was upregulation of only 1 gene while there was downregulation of 56 genes. Apoptosis in the monolayer cultured cells was induced via both the intrinsic and extrinsic apoptotic pathways. However, in the multicellular tumour spheroids (250 and 500 µm) the apoptotic pathway that was followed was not conclusive.

Speaker
Biography:

Amer Elias has his expertise in the genome editing and genome engineering filed. He established a model system intended to catalyze a reaction of DNA fragment replacement in the human genome known as Recombinase Mediated Cassette Exchange (RMCE) that is based on the site specific recombination system of the coliphage HK022 Integrase on purpose to develop a gene therapy method to cure human deleterious mutations. As well Amer is developing a specific anti-cancer binary system based on site-specific recombination for targeting tumors with specific suicide gene in animal models, demonstrating it highly specificity and safety.

Abstract:

Gene therapy is a promising tool for cancer therapeutics. However, a major obstacle persists is the lack of specificity of the expressed toxic gene against cancer cells. Binary systems based on site-specific recombination are one of the most effective potential approaches for cancer gene therapy. In these systems, a cancer specific promoter expresses a site-specific recombinase/integrase that in turn controls the expression of a toxin gene. We have developed a new HK022 bacteriophage Integrase (Int) based binary system that activate a Diphtheria toxin (DTA) gene expression specifically in cancer cells (Fig 1.A). The efficiency, and the specificity of the system were assessed in vitro and in vivo in a lung cancer mouse model. The system presents a significant efficiency and specificity in series of criteria. Strikingly, employment of the developed system to treat mice with lung cancer demonstrates significantly increased longevity (Fig.1 B).  The molecular factors that contribute to the system specificity will be described.

Speaker
Biography:

Joshur Ang has completed his Masters of Research (MRes) from University of Manchester and has been working in Agency for Science Technology and Research (A*STAR) Singapore for the past five years.
 

Abstract:

There is a growing therapeutic interest in human Mesenchymal Stromal Cells (hMSCs), owing to their immunomodulatory properties, differentiation potential and multiple cell sources. To meet clinical demands, transition from planar to scalar technologies is vital for production of industrial grade hMSCs. However, existing methods in detecting cellular senescence are poorly aligned with industrial bioprocesses. Herein we report the discovery of a membrane-permeable fluorescent probe, SenezRedTM, which selectively stains live, senescent hMSCs. Senescent fetal bone marrow hMSC was generated by continuous passaging of hMSC to achieve >120 hours of population doubling time. Cellular senescence was validated using cell cycle analysis and beta-galactosidase assay. Utilizing Diversity-Oriented Fluorescence Library Approach (DOFLA), a library of fluorescent small molecules was screened against proliferative and senescent hMSC. This led to the identification of SenezRedTM, a red fluorescent small molecule (Ex/Em: 631/673 nm) that selectively stains live senescent hMSC in both monolayer and microcarrier cultures. The specificity of SenezRedTM was further validated with beta-galactosidase assay and Ki-67 proliferative staining. Staining conditions were optimized to one hour, significantly reducing the staining time compared to conventional senescent assays. Co-staining of SenezRedTM with Mito-Tracker Green suggested the sub-cellular localisation of the probe in mitochondria. However, further work is required to decipher the detailed mechanism on the selective localisation of SenezRedTM. In summary, we have developed a specific senescent hMSC probe that can potentially be used as a rapid screening tool for quality control purposes of hMSC bioprocessing.

 

Speaker
Biography:

I am M.S student in genetic. I have 27 years old. I have done a research a bout gene Dyrk1B and medicine plant and submitted an article on this topic. I have worked in genetic counseling center for a while. It is mentionable that I have done a project in liver transplantation when I was B.S student.

Abstract:

Introduction: Cardiovascular disease (CVD) is main causes of death in the world. Metabolic syndrome is known as insulin resistance syndrome, which leads to atherosclerosis and coronary artery disease. One of the most important genes that may be involved in metabolic syndrome is Dyrk1B. In this study used Anethum on metabolic syndrome by studying gene expression of Dyrk1B. Anethum has been proven to reduce fat and cholesterol.

Methods: First, extract the RNA from differentiated mesenchymal cells and drug treated mesenchymal cells. using real-time PCR method, were measured Dyrk1B gene of expression. Results were analyzed with One way ANOVA method.

Results: Expression was Dyrk1B when subjected to differentiation, 4.34 fold increases   (pvalue=0.0062).  It was also shown that Dyrk1B expression in differentiated cell groups treated with anethum decreased gene expression compare differentiated cell group alone. (Differentiation: 4.343, Anethum 1: 1.838, Anethum 2: 1.064).

Discussion: Anethum has been proven to reduce fat and cholesterol.  This study Anethum reduced

 

Speaker
Biography:

I am a M.S student in genetic. I am 30 yeaars old. I have done a research a bout gene Dyrk1B and Medicine plant and submitted an article on this topic. I have worked in genetic counseling center for a while. My thesis with Dr mehdi Dianatpour ( phD genetics).

Abstract:

Introduction: Zataria multiflora, known as Avishan-e- Shirazi in Persian, plant that grows wild in central and southern Iran. In Iran, Zataria multiflora is used in traditional folk remedies for its antiseptic, pain-relieving and carminative.  Cardiovascular disease (CVD) is the most common cause of death in developed countries and many developing countries. Metabolic syndrome (MS) is a collection of disorders that occur together and increase your risk of developing type 2 diabetes or cardiovascular disease (stroke or heart disease). In addition, recent studies have shown Dyrk1b gene involved in metabolic syndrome.

Methods: mesenchymal cells were grown in Dulbecco’s Modified Eagle Medium (DMEM) supplemented with 10% fetal bovine serum, and 1% pen/strep. After differentiating of mesenchymal cells, cells were treated separately in the presence of Zataria multiflora extract. RNA extraction from mesenchymal cells was performed and Dyrk1b expression levels were examined by real-time PCR method.

Results: Expression was Dyrk1b when subjected to differentiation, 4.34 fold increases (pvalue=0.0062). It was also shown that Dyrk1B expression in differentiated cell groups treated with Zataria (6 µg/ml: Thyme 1, 12 µg/ml: Thyme 2) decreased gene expression compare differentiated cell group alone.

Discussion: This study provides first evidence that Zataria multiflora can reduced DYRK1B expression and it may be used as an effective and safe therapy for treatment in MS patients

Speaker
Biography:

Nitric oxide (NO) is a two atom free radical that plays an important role as a secondary messenger in cell physiological functions and change of its value influences proteins activity and triggering intracellular signaling cascades. Low frequency electromagnetic field (EMF) by targeting the plasma membrane as well as entering force to the ions and small electrical liga nds alters the cell biology such as cell differentiation.  One way of protein activity regulation  is post  translation modifications that changes structure and function of proteins. Nitric oxide is a signaling molecule in cells that high concentration of this free radical modifies proteins after the translation. NO binds to tyrosine and cysteine residues and influence on the enzyme activity and following that cell fate. In this study effect of high and low concentrations of NO and   electromagnetic field as physical inducer were investigated on the histone deacetylase 2 (HDAC2) gene expression and glutathione peroxidase (GPx) enzyme activity in rat bone marrow mesenchymal stem cells. The cell viability examined in different concentrations of nitric oxide too. The results showed high and low concentrations of NO and EMF alter the gene expression of HDAC2 differently than the control group. NO decreases the activity of GPx. Reduction of cell viability by high concentration of NO attended with the decreased gene expression of HDAC2 and GPx activity, preview important role of NO in cell physiology and fate. NO in high concentration can lead apoptosis and cause to differentiate the stem cells.

Abstract:

Nazanin Haghighat has completed her PhD at the age of 29 years from Tarbiat Modares University. She has published 3 papers in reputed journals.

Biography:

Abstract:

Background and objective: There are common genetic diseases in Hail, but the information about it are still limited. The objective of this cross-sectional study was to detect the most common genetic disease and the risk factor of consanguinity on genetic diseases in Hail.

Subjects and methods: The study samples were determined by multistage probability random sampling procedure. Genetic diseases were obtained from 500 papers and electronic questionnaires. The data were analyzed by using SPSS and calculator.

Results: The proportions of genetic diseases were ordered from highest to lowest as follow: Type 1 Diabetes Mellitus (20%), Congenital Heart Disease (9%), breast cancer (8.2%), Down syndrome (6.4%), Sickle cell disease (3.8%), Muscular Dystrophy (1.8%) and Thalassemia (1%). However, 49.6% of sample answered the question were consanguineous. The proportions showed Thalassemia was the highest disease affected by consanguinity relationship with (100%, p= 0.023), followed Sickle cell disease (73.68%, p= 0.032) and breast cancer (34.14%, p= 0.039).

Conclusion: The data suggested that the most common genetic disease in Hail is Type1 Diabetes Mellitus and consanguinity is one of the risk factor on some of these genetic diseases as Thalassemia, Sickle cell disease and breast cancer.