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Talk: Gene transfer and gene therapy (58 min)

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X Navigable Slide Index
  1. Introduction
  2. Talk outline
  3. Genetic therapy: definition
  4. Genetic therapy: goal
  5. Requirements for successful gene therapy
  6. Gene therapy medicinal products
  7. Viral vector systems (1)
  8. Advantages of retrovirus vectors
  9. Gene transfer using retroviral vectors
  10. Blood-forming stem cells as targets
  11. Goals of gene transfer technology using stem cells
  12. Disadvantages of blood stem cells as targets
  13. Historical perspectives
  14. Application in disorders of hematopoiesis
  15. Viral vector systems (2)
  16. Typical gene transfer protocol
  17. Severe Combined Immunodeficiency (SCID) (1)
  18. Severe Combined Immunodeficiency (SCID) (2)
  19. SCID/CID
  20. X-linked SCID (SCID-X1)
  21. Vector and protocol used in Paris and London
  22. Gene correction of X-SCID: French trial
  23. Gene correction of X-SCID: British trial
  24. CD3+ T lymphocytes after gc gene transfer
  25. Characteristics of transduced T cells
  26. TCRVb diversity
  27. ADA-SCID (1)
  28. ADA-SCID (2)
  29. Toxicity of ADA substrates
  30. ADA-SCID: current treatments
  31. Survival following HLA-mismatched transplantation
  32. Gene correction of ADA-SCID (1)
  33. Gene correction of ADA-SCID (2)
  34. Chronic granulomatous disease: CGD (1)
  35. Chronic granulomatous disease: CGD (2)
  36. Chronic granulomatous disease: trials
  37. Chronic granulomatous disease: results (1)
  38. Chronic granulomatous disease: results (2)
  39. Chronic granulomatous disease: infections
  40. CGD: frequency of mappable RIS
  41. Childhood cerebral adrenoleukodystrophy (CCALD)
  42. CCALD: cause, presentation, and treatment
  43. CCALD: a monogenic, brain demyelinating disease
  44. Rationale for gene transfer in CCALD
  45. Supporting data for gene transfer in CCALD
  46. CCALD: evidence of disease stabilization (1)
  47. CCALD: long term reconstitution
  48. Promising clinical data (CCALD)
  49. CCALD: evidence of disease stabilization (2)
  50. CCALD: excellent long term safety data
  51. Wiskott-Aldrich Syndrome (WAS) (1)
  52. Wiskott-Aldrich Syndrome (WAS) (2)
  53. WAS and gene therapy (trial in Italy)
  54. WAS and gene therapy (trial in Germany)
  55. Hematopoietic stem cell gene transfer (WAS) (1)
  56. Hematopoietic stem cell gene transfer (WAS) (2)
  57. Insertional mutagenesis
  58. Leukemia in X-linked SCID post gene transfer
  59. Clonal expansion: early detection by LAM-PCR
  60. Adverse event associated with gene therapy
  61. Retrovirus insertions near transcription start sites
  62. Retroviral insertion near oncogenes (leukemia)
  63. Safer vector design for gene therapy
  64. Gene transfer using a self-inactivating retrovirus
  65. Overall summary
  66. Prospective: new approaches of importance
  67. Thank you
  68. END
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DETAILED SLIDE INDEX

  1. 1. Introduction
  2. 2. Talk outline
  3. 3. Genetic therapy: definition
  4. 4. Genetic therapy: goal
  5. 5. Requirements for successful gene therapy
  6. 6. Gene therapy medicinal products
  7. 7. Viral vector systems (1)
  8. 8. Advantages of retrovirus vectors
  9. 9. Gene transfer using retroviral vectors
  10. 10. Blood-forming stem cells as targets
  11. 11. Goals of gene transfer technology using stem cells
  12. 12. Disadvantages of blood stem cells as targets
  13. 13. Historical perspectives
  14. 14. Application in disorders of hematopoiesis
  15. 15. Viral vector systems (2)
  16. 16. Typical gene transfer protocol
  17. 17. Severe Combined Immunodeficiency (SCID) (1)
  18. 18. Severe Combined Immunodeficiency (SCID) (2)
  19. 19. SCID/CID
  20. 20. X-linked SCID (SCID-X1)
  21. 21. Vector and protocol used in Paris and London
  22. 22. Gene correction of X-SCID: French trial
  23. 23. Gene correction of X-SCID: British trial
  24. 24. CD3+ T lymphocytes after gc gene transfer
  25. 25. Characteristics of transduced T cells
  26. 26. TCRVb diversity
  27. 27. ADA-SCID (1)
  28. 28. ADA-SCID (2)
  29. 29. Toxicity of ADA substrates
  30. 30. ADA-SCID: current treatments
  31. 31. Survival following HLA-mismatched transplantation
  32. 32. Gene correction of ADA-SCID (1)
  33. 33. Gene correction of ADA-SCID (2)
  34. 34. Chronic granulomatous disease: CGD (1)
  35. 35. Chronic granulomatous disease: CGD (2)
  36. 36. Chronic granulomatous disease: trials
  37. 37. Chronic granulomatous disease: results (1)
  38. 38. Chronic granulomatous disease: results (2)
  39. 39. Chronic granulomatous disease: infections
  40. 40. CGD: frequency of mappable RIS
  41. 41. Childhood cerebral adrenoleukodystrophy (CCALD)
  42. 42. CCALD: cause, presentation, and treatment
  43. 43. CCALD: a monogenic, brain demyelinating disease
  44. 44. Rationale for gene transfer in CCALD
  45. 45. Supporting data for gene transfer in CCALD
  46. 46. CCALD: evidence of disease stabilization (1)
  47. 47. CCALD: long term reconstitution
  48. 48. Promising clinical data (CCALD)
  49. 49. CCALD: evidence of disease stabilization (2)
  50. 50. CCALD: excellent long term safety data
  51. 51. Wiskott-Aldrich Syndrome (WAS) (1)
  52. 52. Wiskott-Aldrich Syndrome (WAS) (2)
  53. 53. WAS and gene therapy (trial in Italy)
  54. 54. WAS and gene therapy (trial in Germany)
  55. 55. Hematopoietic stem cell gene transfer (WAS) (1)
  56. 56. Hematopoietic stem cell gene transfer (WAS) (2)
  57. 57. Insertional mutagenesis
  58. 58. Leukemia in X-linked SCID post gene transfer
  59. 59. Clonal expansion: early detection by LAM-PCR
  60. 60. Adverse event associated with gene therapy
  61. 61. Retrovirus insertions near transcription start sites
  62. 62. Retroviral insertion near oncogenes (leukemia)
  63. 63. Safer vector design for gene therapy
  64. 64. Gene transfer using a self-inactivating retrovirus
  65. 65. Overall summary
  66. 66. Prospective: new approaches of importance
  67. 67. Thank you
  68. 68. END

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TALK'S CITATION

Williams, D.A. (2015), "Gene transfer and gene therapy", in Naldini, L. (ed.), Gene Transfer and Gene Therapy, The Biomedical & Life Sciences Collection, Henry Stewart Talks Ltd, London (online at http://hstalks.com/?t=BL1843836)

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ABOUT THIS TALK

Speaker(s)

Dr. David A. Williams Show Biography

SPEAKER BIOGRAPHY

Dr. David A. Williams – Boston Children’s Hospital, USA

Dr. David A. Williams is Chief of Hematology/Oncology, and Director of Clinical and Translational Research at BCH and the Leland Fikes Professor of Pediatrics at Harvard Medical School and actively cares for children with non-malignant hematology diagnosis. He has served as PI, co-PI or sponsor of five human gene transfer trials in the past 15 years, is currently sponsor or PI of four other trials and has been active in gene transfer research since 1982. He was Editor-in-Chief of Molecular Therapy the leading journal of gene transfer from 2005-2010, and served on the NIH Recombinant DNA Advisory Committee from 2007-2010 and the NIH Gene Therapy Safety Assessment Board from 2009-2010.

Publication Date

March, 2015

Topics Covered

Definition of gene therapy... more

TOPICS COVERED IN THIS TALK

  • Definition of gene therapy
  • Requirements for successful gene therapy
  • Vector systems with application for blood diseases
  • Experience in clinical trials
  • Insertional mutagenesis
  • Prospects and new approaches

Series

Gene Transfer and Gene Therapy

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PRINCIPLES AND GENERAL THEMES
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  • Properties & limitations of transfer vector
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TOPICS COVERED IN THIS TALK

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  • Regulatory considerations
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MAJOR GENE TRANSFER PLATFORMS AND GENE THERAPY STRATEGIES
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  • Virotherapy against tumour cells
  • Virotherapy studies with wild-type vs. genetically-engineered viruses (Adenovirus, Vaccinia & Herpes)
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NEW TECHNOLOGIES FOR SEQUENCE-SPECIFIC EDITING OF GENE EXPRESSION
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Prof. Beverly L. Davidson

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  • Huntington’s disease
  • Vectors for delivering genetic material to brain
  • AAVs for delivering RNAi for reducing gene expression
  • Artificial miRNAs
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  • HTT suppression
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