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    Gene Therapy

    Gene Therapy: Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.

    Webinars

    • OCT 03, 2018 | 12:00 PM
      Mechanisms of CRISPR-Cas Systems: From Adaptive Immunity to Biotechnology
      Mechanisms of CRISPR-Cas Systems: From Adaptive Immunity to Biotechnology
      All forms of life require immune systems to stave off infection from viruses and other pathogens. In bacteria and archaea, clustered regularly interspaced short palindromic repeats (CRISPR) a...
      Speaker: Dipali Sashital, PhD
      Presented at: CRISPR Virtual Event Series 2018
      3
      OCT 03, 2018 | 10:30 AM
      Integrating Novel Advances in Gene Delivery and Genome Engineering for Therapeutic Application
      Integrating Novel Advances in Gene Delivery and Genome Engineering...
      There have been an increasing number of successful human gene therapy clinical trials, and in particular gene delivery vehicles or vectors based on the adeno-associated virus (AAV) have enabl...
      Speaker: David Schaffer, PhD
      Presented at: CRISPR Virtual Event Series 2018
      2
      OCT 03, 2018 | 9:00 AM
      Design, Edit, Analyze and Maximize Your CRISPR-Cas9 Editing Efficiency
      Design, Edit, Analyze and Maximize Your CRISPR-Cas9 Editing Efficiency
      Learn how to optimize CRISPR-Cas9 editing efficiencies in cell lines and primary cell types using modified synthetic sgRNA. Includes tips for achieving maximum knockout and knock-in efficienc...
      Speaker: Kevin Holden, PhD
      Presented at: CRISPR Virtual Event Series 2018
      Sponsored By: Synthego
      2
      OCT 03, 2018 | 9:00 AM
      CRISPRseek and GUIDEseq for Design of Target-Specific Guide RNAs in CRISPR-Cas9 Genome-Editing Systems
      CRISPRseek and GUIDEseq for Design of Target-Specific Guide RNAs...
      The most recently developed genome editing system, CRISPR-Cas9 has greater inherent flexibility than prior programmable nuclease platforms. Because of its simplicity and efficacy, this techno...
      Speaker: Lihua Julie Zhu, PhD
      Presented at: CRISPR Virtual Event Series 2018
      3
      OCT 03, 2018 | 7:30 AM
      Keynote Presentation: Measurements and Standards to Support Confidence in Genome Editing
      Keynote Presentation: Measurements and Standards to Support Confidence...
      The National Institute of Standards and Technology (NIST), is the measurement institute of the U.S., supplying unbiased expertise in measurements and standards to support growth in all sector...
      Speaker: Samantha Maragh, PhD
      Presented at: CRISPR Virtual Event Series 2018
      2
      OCT 03, 2018 | 6:00 AM
      Identification of Pre-existing Adaptive Immune Responses to Cas9 in Humans
      Identification of Pre-existing Adaptive Immune Responses to Cas9...
      The CRISPR-Cas9 system has proven to be a powerful tool for genome editing allowing for the precise modification of specific DNA sequences within a cell. Many efforts are currently underway t...
      Speaker: Carsten Charlesworth, PhD
      Presented at: CRISPR Virtual Event Series 2018
      2
      OCT 01, 2018 | 12:00 AM
      Efficient generation of pancreatic β cell precursors from human pluripotent stem cells
      Efficient generation of pancreatic β cell precursors from human...
      Pancreatic β cell replacement therapy is considered as a potential strategy to treat diabetes. To date, transplantation of pancreatic islets from cadavers is the most effective approach...
      Speaker: Essam Abdelalim
      Presented at: 6th annual 24 Hours of Stem Cellsâ„¢ virtual event
      4
      SEP 18, 2018 | 8:00 AM
      High-throughput CRISPR-Cas9 Genome Engineering in Primary T Cells
      High-throughput CRISPR-Cas9 Genome Engineering in Primary T Cells
      DATE: September 18,2018TIME: 08:00am PDT, 11:00am EDT CRISPR-Cas9 gene editing strategies have revolutionized our ability to engineer the genomes of diverse cell types and sp...
      Speaker: Judd Hultquist, PhD
      Sponsored By: Horizon Discovery
      7
      SEP 01, 2018 | 8:00 AM
      Thermo Scientific Cell Locker System Segregates Stem Cells, Protecting from Contamination and Enhancing Environmental Stability
      Thermo Scientific Cell Locker System Segregates Stem Cells, Protecting...
      Delicate cultures including stem cells and primary cells are increasingly used in cell therapy research and development for applications in oncology, immunology, neurology and more. Commonly,...
      Speaker: Mary Kay Bates, M.S.
      Presented at: 6th annual 24 Hours of Stem Cellsâ„¢ virtual event
      3
      AUG 15, 2018 | 8:00 AM
      3D Spheroid Cell Models for Drug-Resistant Cancer Research
      3D Spheroid Cell Models for Drug-Resistant Cancer Research
      DATE: August 15, 2018TIME: 08:00AM PDT, 11:00AM EDTThe failure of current chemotherapeutic strategies in the fight against cancer can be largely attributed to the occurrence of drug res...
      Speaker: Clarissa Willers, PhD
      Sponsored By: Thermo Fisher Scientific/Gibco
      9 2
      AUG 07, 2018 | 8:00 AM
      Using Reference Standards to Validate your NGS Oncology Workflow
      Using Reference Standards to Validate your NGS Oncology Workflow
      DATE: August 7, 2018TIME: 08:00AM PDT, 11:00AM EDTWe are currently at an exciting precipice in clinical oncology management, where an increased rate of biomarker identification is drivi...
      Speaker: Hannah Child, PhD
      Sponsored By: Horizon Discovery
      10 1
      JUL 19, 2018 | 9:00 AM
      Investigating the role of microRNAs in pancreatic cancer progression
      Investigating the role of microRNAs in pancreatic cancer progression
      DATE:  July 19, 2018TIME:  09:00am PDT Pancreatic ductal adenocarcinoma (PDAC) is a deadly disease with a 5 year-survival rate of approximately 6%. Despite recent...
      Speaker: Silvia Ottaviani, PhD
      Sponsored By: Thermo Fisher Scientific/Gibco
      9 5
      JUN 29, 2018 | 9:00 AM
      Diet and gene interactions in the control of central cell metabolism
      Diet and gene interactions in the control of central cell metabolism
      DATE: June 29, 2018TIME: 09:00AM PDT, 12:00PM EDT There is significant epidemiological evidence to suggest that the consumption of a high-broccoli diet is associated with a r...
      Speaker: Sophie Prosolek
      Sponsored By: Thermo Fisher Scientific/Gibco
      6 2
      JUN 21, 2018 | 1:30 PM
      Pharmacogenetics in Psychiatry
      Pharmacogenetics in Psychiatry
      There is robust evidence supporting strong association with CYP450 (Phase 1) enzymes and influence on drug levels (affecting response & side effects in many cases) with many psychotropic...
      Speaker: Daniel Mueller, PhD
      Presented at: Precision Medicine Virtual Event Series 2018
      1
      JUN 21, 2018 | 12:00 PM
      Whole Genome Sequencing As A Valuable Clinical Tool For the Management of Cancer Patients
      Whole Genome Sequencing As A Valuable Clinical Tool For the Management...
      Advances in DNA sequencing, based upon massively parallel sequencing, has resulted in dramatic advances in DNA sequence output in the past few years. It is now possible to generate terrabases...
      Speaker: David Smith, PhD
      Presented at: Precision Medicine Virtual Event Series 2018
      1
      JUN 21, 2018 | 10:30 AM
      Setting Up Next-Generation Sequencing for the Needs of Routine Molecular Diagnostics With a Focus on the Suitability of the GeneReader NGS System for Lung Cancer Patients
      Setting Up Next-Generation Sequencing for the Needs of Routine...
      The introduction of targeted therapies into the clinical management of lung adenocarcinoma has had a massive impact on patient care. Multiple driver mutations are now druggable and treatments...
      Speaker: Paul Hofman, MD, PhD
      Presented at: Precision Medicine Virtual Event Series 2018
      Sponsored By: QIAGEN
      1
      JUN 21, 2018 | 9:00 AM
      Can AI Beat Cancer?
      Can AI Beat Cancer?
      Finding effective treatments for cancer is fundamentally  a high-dimensional probabilistic planning, search, and optimization problem, characterized by  thousands of molecular subty...
      Speaker: Marty Tenenbaum, PhD
      Presented at: Precision Medicine Virtual Event Series 2018
      1
      JUN 21, 2018 | 7:30 AM
      Keynote Presentation: Paving the Path Towards Precision Medicine in Inflammatory Bowel Diseases
      Keynote Presentation: Paving the Path Towards Precision Medicine...
      Crohn’s disease and ulcerative colitis also known as inflammatory bowel diseases or IBD, are characterized by chronic, recurrent inflammation of the gastrointestinal tract, which result...
      Speaker: Andres Hurtado-Lorenzo, PhD
      Presented at: Precision Medicine Virtual Event Series 2018
      1
      JUN 21, 2018 | 6:00 AM
      Rapid Whole Genome Sequencing in the NICU and PICU Decreases Infant Morbidity and Mortality
      Rapid Whole Genome Sequencing in the NICU and PICU Decreases Infant...
      Rare disease affects 30 million people in the United States, with nearly 50% of cases affecting children. Because nearly 80% of rare diseases are genetic, advancements in genomic testing are...
      Speaker: Stephen Kingsmore, PhD, MD
      Presented at: Precision Medicine Virtual Event Series 2018
      1
      JUN 20, 2018 | 1:30 PM
      Regulation of Precision Medicine
      Regulation of Precision Medicine
      Regulation of precision medicine is rapidly evolving.  The pre-existing regulatory framework did not contemplate many of the products and technologies which are becoming available with p...
      Speaker: Neil Belson
      Presented at: Precision Medicine Virtual Event Series 2018
      3
      JUN 20, 2018 | 12:00 PM
      Precision Medicine: The Way Forward!
      Precision Medicine: The Way Forward!
      Recent advancements in the precision medicine field have presented a great promise to provide innovative solutions to pharmaceutical and healthcare industry to help transform medicine. By fin...
      Speaker: Prasun Mishra, MsC, PhD
      Presented at: Precision Medicine Virtual Event Series 2018
      1
      JUN 20, 2018 | 10:30 AM
      A Pharmacogenomic Approach to Medical Cannabis
      A Pharmacogenomic Approach to Medical Cannabis
      The therapeutic applications of medical cannabis and cannabinoid drugs is controversial, however the number of medical conditions in which cannabinoids and cannabis have shown promise as ther...
      Speaker: Jahan Marcu, PhD
      Presented at: Precision Medicine Virtual Event Series 2018
      1
      JUN 20, 2018 | 10:30 AM
      Panel Discussion: VA Genomics - The Clinic and Research
      Panel Discussion: VA Genomics - The Clinic and Research
      With nearly 9 million covered lives serviced through over 150 medical centers, the VA is one of the largest, unified Healthcare System within the United States. Embedded within this system is...
      Speaker: Laurence Meyer, MD, PhD , Ronald Przygodzki, MD
      Presented at: Precision Medicine Virtual Event Series 2018
      2
      JUN 20, 2018 | 9:00 AM
      Better Care with Better Knowledge
      Better Care with Better Knowledge
      When the human genome was sequenced almost 15 years ago, it was a milestone for the understanding of an individual’s genome.  However to fully understand a disease, millions of ind...
      Speaker: Rupert Yip, PhD
      Presented at: Precision Medicine Virtual Event Series 2018
      Sponsored By: QIAGEN
      1
    OCT 03, 2018 | 12:00 PM
    Mechanisms of CRISPR-Cas Systems: From Adaptive Immunity to Biotechnology
    Mechanisms of CRISPR-Cas Systems: From Adaptive Immunity to Biotechnology
    All forms of life require immune systems to stave off infection from viruses and other pathogens. In bacteria and archaea, clustered regularly interspaced short palindromic repeats (CRISPR) a...
    Speaker: Dipali Sashital, PhD
    Presented at: CRISPR Virtual Event Series 2018
    3
    OCT 03, 2018 | 10:30 AM
    Integrating Novel Advances in Gene Delivery and Genome Engineering for Therapeutic Application
    Integrating Novel Advances in Gene Delivery and Genome Engineering...
    There have been an increasing number of successful human gene therapy clinical trials, and in particular gene delivery vehicles or vectors based on the adeno-associated virus (AAV) have enabl...
    Speaker: David Schaffer, PhD
    Presented at: CRISPR Virtual Event Series 2018
    2
    OCT 03, 2018 | 9:00 AM
    Design, Edit, Analyze and Maximize Your CRISPR-Cas9 Editing Efficiency
    Design, Edit, Analyze and Maximize Your CRISPR-Cas9 Editing Efficiency
    Learn how to optimize CRISPR-Cas9 editing efficiencies in cell lines and primary cell types using modified synthetic sgRNA. Includes tips for achieving maximum knockout and knock-in efficienc...
    Speaker: Kevin Holden, PhD
    Presented at: CRISPR Virtual Event Series 2018
    Sponsored By: Synthego
    2
    OCT 03, 2018 | 9:00 AM
    CRISPRseek and GUIDEseq for Design of Target-Specific Guide RNAs in CRISPR-Cas9 Genome-Editing Systems
    CRISPRseek and GUIDEseq for Design of Target-Specific Guide RNAs...
    The most recently developed genome editing system, CRISPR-Cas9 has greater inherent flexibility than prior programmable nuclease platforms. Because of its simplicity and efficacy, this techno...
    Speaker: Lihua Julie Zhu, PhD
    Presented at: CRISPR Virtual Event Series 2018
    3
    OCT 03, 2018 | 7:30 AM
    Keynote Presentation: Measurements and Standards to Support Confidence in Genome Editing
    Keynote Presentation: Measurements and Standards to Support Confidence...
    The National Institute of Standards and Technology (NIST), is the measurement institute of the U.S., supplying unbiased expertise in measurements and standards to support growth in all sector...
    Speaker: Samantha Maragh, PhD
    Presented at: CRISPR Virtual Event Series 2018
    2
     
     
  • OCT 03, 2018 | 6:00 AM
    Identification of Pre-existing Adaptive Immune Responses to Cas9 in Humans
    Identification of Pre-existing Adaptive Immune Responses to Cas9...
    The CRISPR-Cas9 system has proven to be a powerful tool for genome editing allowing for the precise modification of specific DNA sequences within a cell. Many efforts are currently underway t...
    Speaker: Carsten Charlesworth, PhD
    Presented at: CRISPR Virtual Event Series 2018
    2
    OCT 01, 2018 | 12:00 AM
    Efficient generation of pancreatic β cell precursors from human pluripotent stem cells
    Efficient generation of pancreatic β cell precursors from human...
    Pancreatic β cell replacement therapy is considered as a potential strategy to treat diabetes. To date, transplantation of pancreatic islets from cadavers is the most effective approach...
    Speaker: Essam Abdelalim
    Presented at: 6th annual 24 Hours of Stem Cellsâ„¢ virtual event
    4
    SEP 18, 2018 | 8:00 AM
    High-throughput CRISPR-Cas9 Genome Engineering in Primary T Cells
    High-throughput CRISPR-Cas9 Genome Engineering in Primary T Cells
    DATE: September 18,2018TIME: 08:00am PDT, 11:00am EDT CRISPR-Cas9 gene editing strategies have revolutionized our ability to engineer the genomes of diverse cell types and sp...
    Speaker: Judd Hultquist, PhD
    Sponsored By: Horizon Discovery
    7
    SEP 01, 2018 | 8:00 AM
    Thermo Scientific Cell Locker System Segregates Stem Cells, Protecting from Contamination and Enhancing Environmental Stability
    Thermo Scientific Cell Locker System Segregates Stem Cells, Protecting...
    Delicate cultures including stem cells and primary cells are increasingly used in cell therapy research and development for applications in oncology, immunology, neurology and more. Commonly,...
    Speaker: Mary Kay Bates, M.S.
    Presented at: 6th annual 24 Hours of Stem Cellsâ„¢ virtual event
    3
    AUG 15, 2018 | 8:00 AM
    3D Spheroid Cell Models for Drug-Resistant Cancer Research
    3D Spheroid Cell Models for Drug-Resistant Cancer Research
    DATE: August 15, 2018TIME: 08:00AM PDT, 11:00AM EDTThe failure of current chemotherapeutic strategies in the fight against cancer can be largely attributed to the occurrence of drug res...
    Speaker: Clarissa Willers, PhD
    Sponsored By: Thermo Fisher Scientific/Gibco
    9 2
     
     
  • AUG 07, 2018 | 8:00 AM
    Using Reference Standards to Validate your NGS Oncology Workflow
    Using Reference Standards to Validate your NGS Oncology Workflow
    DATE: August 7, 2018TIME: 08:00AM PDT, 11:00AM EDTWe are currently at an exciting precipice in clinical oncology management, where an increased rate of biomarker identification is drivi...
    Speaker: Hannah Child, PhD
    Sponsored By: Horizon Discovery
    10 1
    JUL 19, 2018 | 9:00 AM
    Investigating the role of microRNAs in pancreatic cancer progression
    Investigating the role of microRNAs in pancreatic cancer progression
    DATE:  July 19, 2018TIME:  09:00am PDT Pancreatic ductal adenocarcinoma (PDAC) is a deadly disease with a 5 year-survival rate of approximately 6%. Despite recent...
    Speaker: Silvia Ottaviani, PhD
    Sponsored By: Thermo Fisher Scientific/Gibco
    9 5
    JUN 29, 2018 | 9:00 AM
    Diet and gene interactions in the control of central cell metabolism
    Diet and gene interactions in the control of central cell metabolism
    DATE: June 29, 2018TIME: 09:00AM PDT, 12:00PM EDT There is significant epidemiological evidence to suggest that the consumption of a high-broccoli diet is associated with a r...
    Speaker: Sophie Prosolek
    Sponsored By: Thermo Fisher Scientific/Gibco
    6 2
    JUN 21, 2018 | 1:30 PM
    Pharmacogenetics in Psychiatry
    Pharmacogenetics in Psychiatry
    There is robust evidence supporting strong association with CYP450 (Phase 1) enzymes and influence on drug levels (affecting response & side effects in many cases) with many psychotropic...
    Speaker: Daniel Mueller, PhD
    Presented at: Precision Medicine Virtual Event Series 2018
    1
    JUN 21, 2018 | 12:00 PM
    Whole Genome Sequencing As A Valuable Clinical Tool For the Management of Cancer Patients
    Whole Genome Sequencing As A Valuable Clinical Tool For the Management...
    Advances in DNA sequencing, based upon massively parallel sequencing, has resulted in dramatic advances in DNA sequence output in the past few years. It is now possible to generate terrabases...
    Speaker: David Smith, PhD
    Presented at: Precision Medicine Virtual Event Series 2018
    1
     
     
  • JUN 21, 2018 | 10:30 AM
    Setting Up Next-Generation Sequencing for the Needs of Routine Molecular Diagnostics With a Focus on the Suitability of the GeneReader NGS System for Lung Cancer Patients
    Setting Up Next-Generation Sequencing for the Needs of Routine...
    The introduction of targeted therapies into the clinical management of lung adenocarcinoma has had a massive impact on patient care. Multiple driver mutations are now druggable and treatments...
    Speaker: Paul Hofman, MD, PhD
    Presented at: Precision Medicine Virtual Event Series 2018
    Sponsored By: QIAGEN
    1
    JUN 21, 2018 | 9:00 AM
    Can AI Beat Cancer?
    Can AI Beat Cancer?
    Finding effective treatments for cancer is fundamentally  a high-dimensional probabilistic planning, search, and optimization problem, characterized by  thousands of molecular subty...
    Speaker: Marty Tenenbaum, PhD
    Presented at: Precision Medicine Virtual Event Series 2018
    1
    JUN 21, 2018 | 7:30 AM
    Keynote Presentation: Paving the Path Towards Precision Medicine in Inflammatory Bowel Diseases
    Keynote Presentation: Paving the Path Towards Precision Medicine...
    Crohn’s disease and ulcerative colitis also known as inflammatory bowel diseases or IBD, are characterized by chronic, recurrent inflammation of the gastrointestinal tract, which result...
    Speaker: Andres Hurtado-Lorenzo, PhD
    Presented at: Precision Medicine Virtual Event Series 2018
    1
    JUN 21, 2018 | 6:00 AM
    Rapid Whole Genome Sequencing in the NICU and PICU Decreases Infant Morbidity and Mortality
    Rapid Whole Genome Sequencing in the NICU and PICU Decreases Infant...
    Rare disease affects 30 million people in the United States, with nearly 50% of cases affecting children. Because nearly 80% of rare diseases are genetic, advancements in genomic testing are...
    Speaker: Stephen Kingsmore, PhD, MD
    Presented at: Precision Medicine Virtual Event Series 2018
    1
    JUN 20, 2018 | 1:30 PM
    Regulation of Precision Medicine
    Regulation of Precision Medicine
    Regulation of precision medicine is rapidly evolving.  The pre-existing regulatory framework did not contemplate many of the products and technologies which are becoming available with p...
    Speaker: Neil Belson
    Presented at: Precision Medicine Virtual Event Series 2018
    3
     
     
  • JUN 20, 2018 | 12:00 PM
    Precision Medicine: The Way Forward!
    Precision Medicine: The Way Forward!
    Recent advancements in the precision medicine field have presented a great promise to provide innovative solutions to pharmaceutical and healthcare industry to help transform medicine. By fin...
    Speaker: Prasun Mishra, MsC, PhD
    Presented at: Precision Medicine Virtual Event Series 2018
    1
    JUN 20, 2018 | 10:30 AM
    A Pharmacogenomic Approach to Medical Cannabis
    A Pharmacogenomic Approach to Medical Cannabis
    The therapeutic applications of medical cannabis and cannabinoid drugs is controversial, however the number of medical conditions in which cannabinoids and cannabis have shown promise as ther...
    Speaker: Jahan Marcu, PhD
    Presented at: Precision Medicine Virtual Event Series 2018
    1
    JUN 20, 2018 | 10:30 AM
    Panel Discussion: VA Genomics - The Clinic and Research
    Panel Discussion: VA Genomics - The Clinic and Research
    With nearly 9 million covered lives serviced through over 150 medical centers, the VA is one of the largest, unified Healthcare System within the United States. Embedded within this system is...
    Speaker: Laurence Meyer, MD, PhD , Ronald Przygodzki, MD
    Presented at: Precision Medicine Virtual Event Series 2018
    2
    JUN 20, 2018 | 9:00 AM
    Better Care with Better Knowledge
    Better Care with Better Knowledge
    When the human genome was sequenced almost 15 years ago, it was a milestone for the understanding of an individual’s genome.  However to fully understand a disease, millions of ind...
    Speaker: Rupert Yip, PhD
    Presented at: Precision Medicine Virtual Event Series 2018
    Sponsored By: QIAGEN
    1
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