Gene Therapy
This learning journey is designed to introduce gene therapy, and the approaches and strategies in it. Three methods of gene transfer are discussed. Specific examples of gene therapy as well as regulatory considerations are also discussed in this journey.
Table of Contents
- Gene Therapy: Basics and Approaches
- Gene Therapy: Milestones and Strategies
- Physical and Chemical Methods of Gene Transfer
- Biological Methods of Gene Transfer: Overview of Viral Vectors
- Review of Gene Therapy Clinical Trials
- Gene Therapy for Choroideremia
- Gene Therapy for Haemophilia
- mRNA for Progeria
- Gene Silencing and Gene Repair
- CRISPR Editing for Duchenne Muscular Dystrophy
- Challenges in the Development of Gene Therapy
- Challenges in the Manufacture of Gene Therapy
- Regulatory Considerations for Gene Therapy Products - US
- Regulatory Considerations for Gene Therapy Products - Europe
1. Gene Therapy: Basics and Approaches
By Dr. Amit Khurana – University Hospital RWTH Aachen, Germany
An excerpt from Gene therapy: basics and therapeutic applications
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2. Gene Therapy: Milestones and Strategies
By Dr. Takis Athanasopoulos – GSK, UK
An excerpt from Recent advances in the development of gene delivery technologies
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3. Physical and Chemical Methods of Gene Transfer
By Prof. Esam Yahya – Universiti Sains Malaysia, Malaysia
An excerpt from Delivery of genes and nucleotides
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4. Biological Methods of Gene Transfer: Overview of Viral Vectors
By Prof. Esam Yahya – Universiti Sains Malaysia, Malaysia
An excerpt from Delivery of genes and nucleotides
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Play Biological Methods of Gene Transfer: Overview of Viral Vectors
5. Review of Gene Therapy Clinical Trials
By Dr. Takis Athanasopoulos – GSK, UK
An excerpt from Recent advances in the development of gene delivery technologies
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6. Gene Therapy for Choroideremia
By Prof. Ian M. MacDonald – University of Alberta, Canada
An excerpt from Choroideremia: a unique retinopathy
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7. Gene Therapy for Haemophilia
By Prof. Roland W. Herzog – Indiana University, USA
An excerpt from Immune tolerance induction to factor VIII
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8. mRNA for Progeria
By Prof. John P. Cooke – Houston Methodist Research Institute, USA
An excerpt from RNA therapeutics: clinical applications and methods of delivery
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9. Gene Silencing and Gene Repair
By Prof. Nicola Brunetti-Pierri – The Telethon Institute of Genetics and Medicine, Italy
An excerpt from Liver-directed gene therapy
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10. CRISPR Editing for Duchenne Muscular Dystrophy
By Prof. Dongsheng Duan – University of Missouri, USA
An excerpt from CRISPR editing therapy for Duchenne Muscular Dystrophy 1
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11. Challenges in the Development of Gene Therapy
By Prof. Mark Lowdell – University College London, UK
An excerpt from ATMPs from academia to industry getting it right first time (GIRFT)
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12. Challenges in the Manufacture of Gene Therapy
By Prof. Mark Lowdell – University College London, UK
An excerpt from ATMPs from academia to industry getting it right first time (GIRFT)
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13. Regulatory Considerations for Gene Therapy Products - US
By Dr. Larissa Lapteva – Center for Biologics Evaluation and Research, FDA, USA
An excerpt from Development and regulation of cellular and gene therapy products: FDA perspective
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14. Regulatory Considerations for Gene Therapy Products - Europe
By Dr. Ana Hidalgo-Simon – European Medicines Agency, The Netherlands
An excerpt from Regulation of ATMPs in Europe: present and future
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