Summary
Cel-, gen- en weefseltechnologie samenvatting
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Dit is een samenvatting te gebruiken voor het vak cel-, gen- en weefseltherapie (ATMP) binnen de master farmaceutische wetenschappen aan de VUB.
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Cel-gen en weefseltherapie:Inhoudstabel
A. Hoorcolleges .............................................................................................. 5
B. Inleiding .................................................................................................... 5
C. Algemeen .................................................................................................. 5
1.1. Definitie ATMP ....................................................................................... 5
1.2. Hierarchie Europese wetgeving ................................................................ 6
1.3. Classificatie van ATMP’s .......................................................................... 7
1.4. De lange weg naar de markt .................................................................... 8
1.5. Effect van ATMP vs (bio)farmaceutica ......................................................11
1.6. Link met “personalized/ precision medicine” .............................................12
1.7. Niet- klinische uitdagingen voor ATMP’s ...................................................14
1.8. Academische wereld en ATMP ontwikkeling ...............................................15
1.9. ATMP in toekomst .................................................................................17
1.10. Ethische aspecten van ATMP’s ..............................................................17
1.11. Take home messages .........................................................................18
D. Geneesmiddelen voor gentherapie: Het centrale dogma en genregulatie ..........20
1.12. Geschiedenis van de gentherapie .........................................................20
1.13. Het centrale dogma (F. Crick) ..............................................................20
1.13.1. Centrale dogma anno 1970 ...........................................................21
1.13.2. Van gen naar eiwit .......................................................................23
1.13.3. Hoe worden de genen geregeld? ....................................................25
1.13.4. Structuur van eukaryotisch mRNA ..................................................29
1.13.5. Types van mutaties ......................................................................30
1.13.6. Splice site mutaties ......................................................................33
1.14. Epigenetica .......................................................................................34
1.14.1. Aanpassingsvermogen: klassiek beeld en aangepast beeld ................34
1.14.2. De agouti muis ............................................................................35
1.14.3. Een goede opvoeding gaat generaties mee ......................................36
1.14.4. Lamarck......................................................................................38
1.14.5. Structuur van het DNA ..................................................................39
1.14.5.1. Opvouwing van DNA ..................................................................40
1.14.6. DNA en histonen vertonen modificaties ...........................................41
1.14.6.1. Epigenetische regulatie van agouti genen .....................................43
1.14.6.2. Epigenetisch effect van knuffelen.................................................44
1.14.7. Manipulatie van het epigenoom ......................................................45
1.15. Gentherapie ......................................................................................47
,1.15.1. Somatische vs kiemlijn gentherapie ................................................47
1.15.2. Principes van gentherapie .............................................................48
1.15.2.1. Gentherapie: benaderingen ........................................................52
1.15.3. Autosomaal dominante aandoeningen .............................................53
1.15.4. Autosomaal recessieve aandoeningen .............................................55
1.15.5. X-chromosoom gelinkt, recessief ....................................................56
1.15.6. X-chromosoom gelinkt, dominant ...................................................58
1.15.7. Gentherapie: vectoren ..................................................................60
1.15.7.1. Virusfamilies waarvan genvectoren zijn afgeleid ............................60
1.15.7.2. Gentherapie: virale vectoren .......................................................61
1.15.7.3. Gentherapie in de tijd ................................................................63
1.15.8. Retrovirale vectoren .....................................................................63
1.15.8.1. Retrovirussen: taxonomie ...........................................................63
1.15.8.2. Retrovirale structuur ..................................................................64
1.15.8.3. Retrovirale levenscyclus .............................................................67
1.15.8.4. Retrovirale vectoren: plasmiden en productie ................................69
1.15.8.5. Retrovirale vectoren: basisstructuur expressievector ......................72
1.15.8.6. Retrovirale vectoren: pros en cons ..............................................73
1.15.8.7. Aangeboren immuundeficiëntie ziekten ........................................74
1.15.9. Lentivirale vectoren ......................................................................80
1.15.9.1. Lentivirale structuur ...................................................................80
1.15.9.2. Lentivirale vectoren: plasmiden ...................................................81
1.15.9.3. Lentivirale vectoren: productie ....................................................84
1.15.9.4. Lentivirale vectoren: "changing coats"(= pseudotypering) ..............85
1.15.9.5. Lentivirale vectoren: promotorkeuze ............................................86
1.15.9.6. Lentivirale vs. Retrovirale vectoren ..............................................87
1.15.10. Adenovirale vectoren ....................................................................88
1.15.10.1. Adenovirus: structuur en ontwikkeling ........................................88
1.15.10.2. Targeting ................................................................................90
1.15.10.3. Adenovirale vectoren: plasmiden ...............................................90
1.15.10.4. Pros en cons ...........................................................................91
1.15.11. AAV- vectoren .............................................................................92
1.15.11.1. AAV vector: plasmide ...............................................................93
1.15.11.2. AAV- gebaseerde vectoren: serotypes ........................................94
1.15.11.3. AAV- gebaseerde vectoren: pros en cons ....................................94
1.15.11.4. De andere kant van de medaille: de eerste dode tgv gentherapie ..95
1.15.11.5. Glybera: the million dollar drug .................................................95
1.15.11.6. Gene therapy medicinal products (GTMP) ...................................98
, 1.15.11.7. Gentherapie trials en algemene informatie gentherapie ................99
E. Gene editing ........................................................................................... 102
1.16. Inleiding ......................................................................................... 102
1.17. Gene insertion vs gene editing. .......................................................... 103
1.18. Toepassingen van gene editing .......................................................... 104
1.19. Targeted DNA double strand breaks (DSBs) ......................................... 105
1.20. NHEJ vs HR ..................................................................................... 106
1.21. Hoe maken we DSB’s? ...................................................................... 108
1.22. Benaderingen voor gene editing ......................................................... 109
1.22.1. Meganuclease (20-40 bp/ enzyme) .............................................. 109
1.22.2. Zinc fingers (3 bp/finger) ............................................................ 109
1.22.3. TALEN (1 bp/ module) ................................................................ 113
1.22.4. CRISPR Cas (1 bp/base) ............................................................. 114
1.22.5. Cas9 fylogenie ........................................................................... 118
1.22.6. Toepassingen CRISPR/Cas9 ......................................................... 119
1.23. Gene editing: toepassingen in het algemeen. ....................................... 120
1.24. HIV ................................................................................................ 121
1.24.1. Structuur van het HIV virus ......................................................... 121
1.24.2. Routes van HIV transmissie ......................................................... 122
1.24.3. Mechanisme van HIV infectie ....................................................... 123
F. Inleiding immunologie .............................................................................. 127
1.25. Wat is het immuunsysteem? .............................................................. 127
1.26. Witte bloedcellen zijn de belangrijkste immuuncellen van het lichaam ..... 129
1.27. Welke cellen herkennen pathogenen? ................................................. 132
1.28. Fagocytose ...................................................................................... 133
1.29. Kenmerken van inflammatie .............................................................. 133
1.30. De inflammatoire respons ................................................................. 134
1.31. Het aangeboren immuunsysteem stuurt het adaptief immuunsysteem .... 136
1.32. Expansie van specifieke lymfocyten bij infectie .................................... 138
1.33. De B- cel ......................................................................................... 139
1.34. Antilichaam als herkenningspunt voor pathogenen ............................... 140
1.35. De T- cel ......................................................................................... 141
1.36. B-cel en T-cel receptoren .................................................................. 148
1.37. Evolutie van het immuunsysteem ....................................................... 148
1.38. Waar is het immuunsysteem? ............................................................ 149
1.39. Het mucosaal immuunsysteem .......................................................... 149
1.40. Microflora in de menselijke darm ........................................................ 150
1.41. Rol van microbiota en de Europese tegenhanger .................................. 150
, 1.42. Probiotica in het dagelijkse leven ....................................................... 151
1.43. Ilya Mechnikow, de vader van de probiotica ......................................... 151
1.44. Beschermer tegen ziekte maar ook ziekmaker ..................................... 151
1.45. Hygiëne, vaccinatie (en antibiotica) als mijlpalen in de geschiedenis van de
geneeskunde ................................................................................................ 152
1.45.1. Dodental veroorzaakt door historische pandemieën ........................ 153
1.45.2. Antisepsis ................................................................................. 153
1.45.3. Mortaliteit van kraamkoorts ......................................................... 154
1.45.4. Belangrijkste doodsoorzaken toen en nu… ..................................... 154
1.45.5. De pokken................................................................................. 154
1.45.6. Vaccinatie ................................................................................. 154
1.46. Kindvaccins ..................................................................................... 156
1.47. Impact van vaccins........................................................................... 157
1.48. Voorbeelden van vaccinatiecontroverses ............................................. 157
1.49. Polio ............................................................................................... 158
1.50. Congenitale rubella........................................................................... 158
1.51. Tetanus .......................................................................................... 158
1.52. Waarom vaccinatie een sociaal gebeuren is ......................................... 158
1.53. Huidige en toekomstige noden voor vaccinatie ..................................... 160
G. Tumor immunology and cancer immunotherapy .......................................... 161
1.54. Tumor immunology "avant la lettre" ................................................... 161
1.55. Birth of immunology ......................................................................... 161
1.56. The first case of cancer immunotherapy .............................................. 163
1.56.1. Contempory view on Coley's toxins .............................................. 170
1.57. Terug in de tijd… .............................................................................. 171
1.58. Coming from the other side: CAR T-cells ............................................. 178
H. Gastlessen .............................................................................................. 203
I. Voorbeeldvragen examen ........................................................................ 232
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