Learn All About Gene Therapy | Gene Therapy Network

Web Name: Learn All About Gene Therapy | Gene Therapy Network

WebSite: http://www.genetherapynetwork.com

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Podcast: The Future of Gene Therapy and Genetic Diseases

Podcast: Gene Therapy in Action: Adeno-Associated Viral Vectors

Newborn & Prenatal Genetic Screening

ResourcesDownloadable ResourcesGlossaryGene Therapy Clinical Trials in the U.S.AboutResourcesDownloadable ResourcesGlossaryGene Therapy Clinical Trials in the U.S.About

Welcome to the leading edge of gene therapy education

The Gene Therapy Network provides the latest, expert-led, educational and practical gene therapy content, based on the needs of healthcare professionals around the world and aligned with professional bodies

MODULE 1

Overview of Gene Therapy

An overview of gene therapy and some of the key concepts in this field

MODULE 2

Monogenic and Complex Diseases

An overview of genetic abnormalities and current treatment strategies for monogenic diseases

MODULE 3

Safety of Gene Therapy

A review of the two types of immune responses in the context of two of the most widely studied viral vectors

MODULE 4

Practical Considerations

An overview of the biological risks and containment practices for use of AAV-based gene therapies

MODULE 5

Gene Replacement

One important approach to gene therapy is gene replacement. This video further explores this important concept.

MODULE 6

Current Therapeutics & Research

A review of the gene therapy development landscape and exploration of marketed agents

NEWS

Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in AustraliaSource: Gene Therapy - Issue - nature.com science feeds | Published: October 2, 2022 - 12:00 amCharacterization of a novel Pde6b-deficient rat model of retinal degeneration and treatment with adeno-associated virus (AAV) gene therapySource: Gene Therapy - Issue - nature.com science feeds | Published: September 29, 2022 - 12:00 amDesign, construction and in vivo functional assessment of a hinge truncated sFLT01Source: Gene Therapy - Issue - nature.com science feeds | Published: September 16, 2022 - 12:00 amChanging trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapiesSource: Gene Therapy - Issue - nature.com science feeds | Published: September 12, 2022 - 12:00 amAdeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheepSource: Gene Therapy - Issue - nature.com science feeds | Published: September 1, 2022 - 12:00 amMultiplex viral tropism assay in complex cell populations with single-cell resolutionSource: Gene Therapy - Issue - nature.com science feeds | Published: August 23, 2022 - 12:00 am

STEERING COMMITTEE

Meet our multidisciplinary, internationally renowned steering committee of experts:

Chris Jenkins, PhD, MPH Chairperson
Principal Partner and Chief Gene Therapy Biosafety Officer at Clinical Biosafety Services

Diana Bharucha-Goebel, MDDirector of the Muscular Dystrophy Association Care Center and Assistant Professor in the Division of Neurology

Arthur Burghes, PhDProfessor at Wexner Medical Center College of Medicine in the Department of Biological Chemistry and Pharmacology

James Dowling, MD, PhD, FANAProfessor of Pediatrics and Molecular Genetics at the University of Toronto

Uta Griesenbach, DIPL-BIOL, PhDProfessor in Molecular Medicine at the National Heart and Lung Institute at Imperial College London

Kristi Jones, PhDClinical Associate Professor of Pediatrics, Child Health and Genetic Medicine at The University of Sydney, and Co-Head of the Department of Clinical Genetics at The Children’s Hospital at Westmead

John Petrich, MS, RPhDirector Investigational Drug Services at the Cleveland Clinic

Website Navigation

Overview of Gene TherapyWhat is Gene TherapyGene Manipulation StrategiesDelivery of Gene TherapyHistory of Gene TherapyOvercoming Challenges in Gene TherapyMonogenic and Complex DiseasesMonogenic DiseasesOverview of Monogenic DiseasesClassifications of Monogenic DiseasesFour Types of Genetic AlterationsTreatment StrategiesTreatment Strategies of Monogenic Diseases – ProteinTreatment Strategies of Monogenic Diseases – SubstrateTreatment Strategies of Monogenic Diseases – RNATreatment Strategies of Monogenic Diseases – DNAComplex DiseasesOverview of Complex DiseasesOverview of CancerTreatment Strategies in CancerSafety of Gene Therapy Immunogenicity of Gene TherapiesImmunogenicity of Lentiviral VectorsImmunogenicity of Adeno-Associated Viral VectorsImpact of the Immune Response on Gene TherapyStrategies to Avoid the Host Immune ResponseImmunogenicity Profiles of Selected Gene TherapiesOverview of Viral Vector SheddingFactors Influencing Vector SheddingAAV Vector Shedding Data in Clinical TrialsPracticalConsiderations FAQ: Considerations for Working with In Vivo Gene TherapiesHandling Gene Therapy in Clinical SettingsProtocols for Handling Gene Therapy ProductsInstitutional Preparedness for Handling In Vivo Gene TherapyReceipt, Storage, Preparation, and Dispensing of In Vivo Gene TherapyTransport and Administration of In Vivo Gene TherapyWaste Disposal and Decontamination Following the Preparation and Administration of In Vivo Gene TherapySpills and Accidental ExposureGene TherapyApproachesGene Replacement TherapyWhat is Gene Replacement TherapyMonogenic Diseases – A Therapeutic Target for Gene Replacement TherapyGene Inhibition TherapyOverview of Gene Inhibition TherapyEndogenous RNAi PathwayTherapeutic Triggers of RNAishRNA Versus Artificial miRNAComponents of Gene Inhibition TherapyGenetic Modulation of Immune CellsGenetic Modulation of Immune Cells: OverviewIntroduction to Genetically Engineered T CellsCAR T-cell Therapy: How Does It WorkTCR Therapy: How Does It WorkGene-Based Vaccines: How Do They WorkCytokine Gene Transfer: How Does it WorkGenome EditingOverview of Genome EditingCRISPR-Cas9 Genome EditingGene AdditionHow Does Gene Addition WorkCurrent Therapeutics & ResearchGene Therapies in Research: OverviewApproved TherapiesExpert Series & PodcastsExpert SeriesMeet the Experts: Preparing Your Institution for Gene TherapyExpert Series: Institutional PreparednessExpert Series: Organizational ChallengesExpert Series: Engineering Controls and Physical FacilitiesExpert Series: Handling and ExposurePodcastsA Brief History of Gene Therapy and the Discovery of DNAHow Does Gene Therapy Work? Correcting Disease-Causing Genetic VariationsThe Future of Gene Therapy and Genetic DiseasesGene Therapy in Action: Adeno-Associated Viral Vectors

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