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  • Resources
    • Downloadable Resources
    • Gene Therapy Clinical Trials in the U.S.
  • About
gtn
  • Overview of Gene Therapy
    • What Is Gene Therapy?
    • Gene Manipulation Strategies
    • Delivery of Gene Therapy
    • History of Gene Therapy
    • Overcoming Challenges in Gene Therapy
  • Monogenic Diseases
    • Overview of Monogenic Diseases
    • Classifications of Monogenic Diseases
    • Four Types of Genetic Alterations
    • Treatment Strategies
      • Treatment Strategies of Monogenic Diseases – Protein
      • Treatment Strategies of Monogenic Diseases – Substrate
      • Treatment Strategies of Monogenic Diseases – RNA
      • Treatment Strategies of Monogenic Diseases – DNA
  • Safety of Gene Therapy
    • Immunogenicity of Gene Therapies
      • Immunogenicity of Lentiviral Vectors
      • Immunogenicity of Adeno-Associated Viral Vectors
      • Impact of the Immune Response on Gene Therapy
      • Strategies to Avoid the Host Immune Response
      • Immunogenicity Profiles of Selected Gene Therapies
    • Overview of Viral Vector Shedding
    • Factors Influencing Vector Shedding
    • AAV Vector Shedding Data in Clinical Trials
  • PracticalConsiderations
    • Webinar: Considerations for the Use of In Vivo AAV-Based Gene Therapies
    • FAQ: Considerations for Working with In Vivo Gene Therapies
    • Handling Gene Therapy in Clinical Settings
    • Protocols for Handling Gene Therapy Products
      • Institutional Preparedness for Handling In Vivo Gene Therapy
      • Receipt, Storage, Preparation, and Dispensing of In Vivo Gene Therapy
      • Transport and Administration of In Vivo Gene Therapy
      • Waste Disposal and Decontamination Following the Preparation and Administration of In Vivo Gene Therapy
      • Spills and Accidental Exposure
  • Gene TherapyApproaches
    • Gene Replacement Therapy
      • What is Gene Replacement Therapy?
      • Monogenic Diseases – A Therapeutic Target for Gene Replacement Therapy
    • Gene Inhibition Therapy
      • Overview of Gene Inhibition Therapy
      • Endogenous RNAi Pathway
      • Therapeutic Triggers of RNAi
      • shRNA Versus Artificial miRNA
      • Components of Gene Inhibition Therapy
  • Current Therapeutics & Research
    • Gene Therapies in Research: Overview
    • Approved Therapies
×
  • Overview of Gene Therapy
    • What Is Gene Therapy?
    • Gene Manipulation Strategies
    • Delivery of Gene Therapy
    • History of Gene Therapy
    • Overcoming Challenges in Gene Therapy
  • Monogenic Diseases
    • Overview of Monogenic Diseases
    • Classifications of Monogenic Diseases
    • Four Types of Genetic Alterations
    • Treatment Strategies
      • Treatment Strategies of Monogenic Diseases – Protein
      • Treatment Strategies of Monogenic Diseases – Substrate
      • Treatment Strategies of Monogenic Diseases – RNA
      • Treatment Strategies of Monogenic Diseases – DNA
  • Safety of Gene Therapy
    • Immunogenicity of Gene Therapies
      • Immunogenicity of Lentiviral Vectors
      • Immunogenicity of Adeno-Associated Viral Vectors
      • Impact of the Immune Response on Gene Therapy
      • Strategies to Avoid the Host Immune Response
      • Immunogenicity Profiles of Selected Gene Therapies
    • Overview of Viral Vector Shedding
    • Factors Influencing Vector Shedding
    • AAV Vector Shedding Data in Clinical Trials
  • PracticalConsiderations
    • Webinar: Considerations for the Use of In Vivo AAV-Based Gene Therapies
    • FAQ: Considerations for Working with In Vivo Gene Therapies
    • Handling Gene Therapy in Clinical Settings
    • Protocols for Handling Gene Therapy Products
      • Institutional Preparedness for Handling In Vivo Gene Therapy
      • Receipt, Storage, Preparation, and Dispensing of In Vivo Gene Therapy
      • Transport and Administration of In Vivo Gene Therapy
      • Waste Disposal and Decontamination Following the Preparation and Administration of In Vivo Gene Therapy
      • Spills and Accidental Exposure
  • Gene TherapyApproaches
    • Gene Replacement Therapy
      • What is Gene Replacement Therapy?
      • Monogenic Diseases – A Therapeutic Target for Gene Replacement Therapy
    • Gene Inhibition Therapy
      • Overview of Gene Inhibition Therapy
      • Endogenous RNAi Pathway
      • Therapeutic Triggers of RNAi
      • shRNA Versus Artificial miRNA
      • Components of Gene Inhibition Therapy
  • Current Therapeutics & Research
    • Gene Therapies in Research: Overview
    • Approved Therapies
Video

The Process of Gene Replacement Therapy

A video showing how gene replacement therapy works
Download
Slides

Gene Therapy Approvals

Summary of approved gene therapy agents
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Video

Recorded Webinar: Practical Considerations for the Use Of In Vivo AAV-Based Gene Therapies

An overview of the biological risks and containment practices for use of AAV-based gene therapies presented by Dr. Chris Jenkins
Download
Slides

GTnetwork Delivery of Gene Therapy

A summary of delivery systems and methods for gene therapy
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Slides

GTnetwork Biosafety Handling for Gene Replacement Therapy

Overview of gene therapy and biosafety handling considerations
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Slides

GTnetwork Gene Therapy Clinical Trials

Disease targets and approved clinical trials by year from 1989–2018
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Slides

GTnetwork Genetic Diseases & Gene Therapy Approaches

Review of targeted disease types and approaches for monogenic diseases
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Slides

Webinar Slides: Practical Considerations for the Use of In Vivo AAV-Based Gene Therapies

An overview of the biological risks and containment practices for use of AAV-based gene therapies presented by Dr. Chris Jenkins [webinar slides]
Download
Slides

GTnetwork Gene Replacement Therapy

Overview of gene replacement therapy as a treatment strategy
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Slides

GTnetwork Gene Manipulation Strategies

Manipulation strategies utilized for gene therapy
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Slides

GTnetwork Gene Therapy Milestones

Timeline of major milestones
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PDF

Webinar Q&A: Practical Considerations for the Use Of In Vivo AAV-Based Gene Therapies

Participant Q&As with Dr. Chris Jenkins from the "Practical Considerations for the Use Of In Vivo AAV-Based Gene Therapies" webinar
Download
PDF

GTnetwork AAV Biosafety Summary

Summary of biosafety considerations related to AAV-based gene therapies
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  • Privacy Policy
  • Support Statement
  • Community Guidelines
  • Contact Us
  • Overview of Gene Therapy
    • What Is Gene Therapy?
    • Gene Manipulation Strategies
    • History of Gene Therapy
    • Delivery of Gene Therapy
    • Overcoming Challenges in Gene Therapy
  • MonogenicDiseases
    • Overview of Monogenic Diseases
    • Four Types of Genetic Alterations
    • Current Treatment Strategies
      • Treatment Strategies of Monogenic Diseases – Protein
      • Treatment Strategies of Monogenic Diseases – Substrate
      • Treatment Strategies of Monogenic Diseases – DNA
      • Treatment Strategies of Monogenic Diseases – RNA
  • Current Therapeutics & Research
    • Gene Therapies in Research: Overview

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