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GRUNDFORSKNING - GENTERAPI AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 GENTERAPI Gene therapy is ‘the use of genes as medicine’. It involves the transfer of a therapeutic or working gene copy into specific cells of an individual in order to repair a faulty gene copy. Thus it may be used to replace a faulty gene, or to introduce a new gene whose function is to cure or to favourably modify the clinical course of a condition. www.esgct.eu AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 GENTERAPI SOM BEHANDLING FOR SCID SCID: Severe combined immunodeficiency Immundefekt Prævalens: 1:100.000 Behandling: knoglemarvstransplantation eller genterapi Bivirkning ved SCID genterapi: leukæmi AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 THE CENTRAL DOGMA OF MOLECULAR BIOLOGY DNA AU AARHUS UNIVERSITET RNA Protein DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 DNA AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 THE HUMAN KARYOTYPE AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 AUTOSOMALE DOMINANT DISEASE AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 HEREDITARY ANGIOEDEMA (HAE) FACTS AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 GENETICS - HAE is caused by mutations in the serping1 gene - These mutations lead to erroneous production of C1INH (Medication??) - C1INH: a serine protease inhibitor in the contact system AU AARHUS UNIVERSITET Chromosome 11 DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 THE CONTACT SYSTEM AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 THE CONTACT SYSTEM Trauma Bradykinin C1INH AU AARHUS UNIVERSITET Edema C1INH DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 THE CONTACT SYSTEM Trauma AU AARHUS UNIVERSITET Bradykinin ↑ Edema DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 TYPES OF HAE (1+2) Type I AU Distribution 85% Mutation Deletions or insertions – throughout whole serping1 C1INH protein level 10-30% of normal C1INH functional level Low AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 TRANS-INHIBITION Chr. 11 normal Serping1 gene DNA RNA Protein Chr. 11 mutated Serping1 gene DNA RNA Protein AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 TRANS-INHIBITION Chr. 11 normal Serping1 gene DNA RNA Chr. 11 mutated Serping1 gene DNA RNA AU AARHUS UNIVERSITET Protein DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN Protein 3. FEBRUAR 2015 MODELS? AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 CELL WORK IN THE LABORATORY AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 HELA CELLER • Immortaliseret cellelinie • Humane livmoderhalskræftceller • Kromosomerne består af humane gener og gener fra HPV virus, som forårsagede kræften • Isoleret fra Henrietta Lack i 1951 Henrietta Lack • Cellelinjen blev dannet af forskeren George Otto Gey på John Hopkins Universitet uden Henriettas viden Født i Virginia i 1920 AU AARHUS UNIVERSITET Fik i 1951 diagnosticeret livmoderhalskræft – døde senere samme år DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 TRANS-INHIBITON?? ???% C1INH AU AARHUS UNIVERSITET ???% C1INH DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 TRANS-INHIBITON?? 100% C1INH AU AARHUS UNIVERSITET 10-20% C1INH DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 FORKØLEDE BAKTERIER OG FREMTIDENS GENTERAPI Bakterier kan inficeres af virus Bakterier har primitivt ’immunforsvar’ CRISPR/Cas9 systemet Gemmer info fra tidligere virus infektioner Genkender virus og klipper dets DNA i stykker AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 HVAD KAN VI BRUGE DET TIL? Omdirigering af enzymet Vi bestemmer hvor DNA’et klippes over Reparation med et DNA ’plaster’ AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 HVAD MED HAE? Målrettet korrektion af HAE mutationer 3.209.289.105 basepar AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 HVAD MED HAE? Kromosom 11 135.006.516 basepar AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 ctgatttacaggaactcacaccagcgatcaatcttccttaatttgtaactgggcagtgtcccgggccagccaatagctaagactgccccccccgcaccccaccctccctgaccctgggggactctctactcagtctgcactggagctgcctggtgaccagaagtttggagtaggtttggtgctgggcaggggtggggagtagggtggaaagcatggagtgaagaggtctagggagggggtctcctcacccccgccttcctgcccgccttgatctcgggggtctctataggcttgcttcc acctgggacttctgcctcctcctaccccagcccctcccgcctcaggcctgttgtgctcagccccccaggacctcccctcccccacgcctctggcctcattgtttggttaaagcaggaccccctccccctcccaccacctcccctccgactgaacagatggacagagacccgggcccacggggagaggaagggccagccggtgccgggaaagggaagcggtttggggaaaacaaaacagagggaggagccagggagaaggtggccccaggagggaggaggagggaattcgcta agagggactggggcctgagacggaatgggggcgggccccgggcggggtgggggcccctgggctcccagggtgggagctggctccgaggctggctggctccgcaggtccgctgacgtcgccgcccagatggcctccaggctgaccctgctgaccctcctgctgctgctgctggctggggtatgtggtcccttgtgggatgggggacgggggtggagacgggaggcgggatggtgcggggtgcgggcggtggctgaggattaacccttcaggctccggggaatgaggagagctcc tcttgggatcattgagtgtgatccttgcacacgcactcgtagatggtggaaagagctcaggactcagacagatgcaagttctaatcttggcgggccatgtaggagctgcgcagcgtggtcctgaggacgtcacttttctcagcctactcctcctgatttataaaatgttgaataaacagatcccaccctgtccaccaagcaggcttggcaggaggatcacatagggcaaggagatgggaagcattttgtgatatcaaagcaagagtttgttgtagaaaagagctgtggggaggaga ggtttggctgctgtgacacagacggtggtcccaggctagggcctgtgaagacagggaagaatggtagcaaacatacgtgggtctgggtgggggtcaatagcagtgagaggtcccagtggtctgggaggtcatggactagaaataaggctaagaagtgcaggatcccaggagaaaggtaggtaggggtgtgtgtatgtgcgtgtgtgtgtgtgtgcacgcgcaagttggtatcacatactcagcagatgatgtagttatgagcagggagtttggaatcagagaccttgttttgac ccctagctgtttctcccatttctgtgagaccctggactagttaatatgtgtcggtatgagtttctctacctgtgaaatggagctaatgcgtggtttctcagtggaggatctgttgacattggggctggataattcattgctgaggggctaccctgcgcattttaggatgttttgcagtatatctctagcctctacgcactagattccggtagcatcttccctgctgtctccctgtgacaaccaaaaatgtctctggacattgccaaatatcccctgggagtcaaaattgtttcagagccgctggc 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aatggcatgaacccgggaggcggagcttgcagtgagccgagattgcgccactgcactccagcctgggcaacagagcgagacaccatctcaaaaaaaaaaaaaaaaaatggggcgggcgggccaggcgcggtgtctcacacctgtaatcccagcactttgggaggctgaggtgggcggatcacttgaggtcaggagttcaagaccagcctggccaacaccaggaaaccctgtctctgttaaaaatacaaaaattagccaggtgtggtggcacgcgcctgtagtcccagcag gagaataacttcaacctgggagacagaggttgcagtcagctgagatcgcaccactgcattccagcctgggtgacagaccgagactctgtctcaaaaaaaaaaaaaaaaagaagaatacccatatgcattcattaatatatagggctagagggctagagagctatagacataaaatagacaaaaaatttttttgctcatttttgggtcaaaggagtcttgggactctaattcttttaatttttgtgttatgtgaatttgttatcatttacatgtattatgttattaagtaggtaataatg ataatactaataataaacttacaaaacgatccaatgtagttgttttcagactttgttcctcggagccctaggactttgcaaagctgtttctggagtcatggtgggagtggggtgtggggagcctgagcagtggggaagggttcaccacttcctgtgagagggaaacggaccagctgggctctgagctccccacccagcctcagccagggtccattttatattgtgggcttcagacatgcctttgtttgaaagcagttctgctgctttaaaatgtttgataaccattgaactaatctaccca 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aatctacttagtcctagaaaaattatctttcaagtgctttttttttttttttttcagtggactgaaggcttttgcagagcaaaagccaggaagttttgtcacaaacttccaccaaggccaggaagcagagtctattccgtgccaaaattaagggaagaaaagagggaaaatctaggcaaatggaatttaatatctcaaaaagattatcttgccgagccatagtcacatagcaagctgaggcagcggaggcttggggctacttctcttgttcttggttctgggtttaccttctttgggcctta tttgccacatctgtaagaggagtgggctggaccgcgctccaccatgccgtattcactaagtgagcagatagaaccatagaaagcatgctcactctcaaatcgtgctcatggaaagaacgacgtgttcaggactcatgcctccctttctcaacatacccccagacctggccataagggacacctttgtgaatgcctctcggaccctgtacagcagcagccccagagtcctaagcaacaacagtgacgccaacttggagctcatcaacacctgggtggccaagaacaccaacaaca agatcagccggctgctagacagtctgccctccgatacccgccttgtcctcctcaatgctatctacctgagtggtaagggtgcccttagccagttagtcttcccattctgggtccttcttcccctcctggcttcaaagcccacttaaccccaagttctacaatcggatctcaatgtccctgcactactctttgctaacaaggcttttagctcctcttcatccttttcctacctgcattagagcaaccctcccacctcttccctctagccaagtggaagacaacatttgatcccaagaaaaccagaa tggaaccctttcacttcaaaaactcagttataaaagtgcccatgatgaatagcaagaagtaccctgtggcccatttcattgaccaaactttgaaagccaaggtaagttcttaacctttccttctcctgtttgaaacctacttgagtctcctgactttttttctgctgtagtcccatcattttggggtacatgcttacaaattcatcacttctactccttccatctgtatttccaccctatcttttctccttctcctttctctagccttggccaaggcagacattgtatattttaggctggaaacaggat tctcaagtttcttcatgcatctctactatattgaatggcaaaatgtgagtcgtgttcctattctacacatctgtttctttttccagtcaactcatcagaagactctggtggcttagcaagtcctgtgtgtatgtgggtacctgtgtacagcatacatatacatgtgtagacgtgtataagtatatccttgtatatgcatatatgtggtaagtgcatgtgtgcacatgagttaaatggcttaagtgcattagcagaaattgaacaatttagtttgatgagtagggagggagagaagacagaata tggagcccagaggatcccaactggcaagctatctcatccccagttaccagatatttccacctttttaggatggcaaaacacagacctatgagacagaaaacagtaagagacaatgtctcaatacctggctgagaaacaaaggctctgttcttggaacatgctaggcttggttccacattgggagctttccttagcttttgtttctgcctgtatccctcttcactcagatcctcacatagcttgttccttcttgtcatttgggtgtcagctcagaggtcaccttggtggagaagtcttctcccc 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actattcgaattataagaacaagtatttcctctctaaaaatgtttttaaagtttcacacaaaactctaaaccaaaaatcattgtaaaaatcccttgggagtagactgttgaaaccattgaggtgaggtgataggttaaggtgagcattctggacatataatatcaataataaatattcaaatacaaagcaggtataatatgaagagtaatttctatctccccaagccaatatgagttttgtttgtttgtttgtttttcttttgagataaagtctcattctgttgcccaggccggagtgcagt ggcacaatcccagctctctgtaacctctgcctcccaggttcaagcgattctcctgccttagcctccctagtagctgggattataggtgcctgccaccactcccagctaatttttgtatttttagtagagatggggtttcaccatgttggccaggctggtcatgaactcctgacctcaagtgctcgtccgtcttcggcctcctaaagtgctggcattacaggcgtgagccactgcacccagcctgagttttttttttttttttaagccaaaggaggaggagtagttttgtgcacaaaagccactat ttagagttccttaggttttaaacagataattaatttaatgtactaacaaatgcaaaattaaaattttacttatgccttttaattttaagctgaacttgctacattaagagtagggtgatttggctgggcgcagtggctcatgcctataatcccagcactttaggaggctgaggcaggtggatcacttgaagctaagagtttgagaccagcctggccaacatggtggaattctgtctctactaaaaatacaaaaattagccaggcatggtggcacatgtctgtaatcccagctactcagga tgctaaggcacagaatcacttgaacctgggaggcagaggttacagtgagccaaaatcgcgccactgcactccaacctgggcaacacagcaagactctgtctcaaaacacaaaaaaagaataggctgggtacagtggctcacgcctgtaatcccagcactttgggaggccgaggcaggtgaatcacctgaggtcaggagttcgagaccagcttggccaacattgtgaaaccccatctccattaaaaatacaaaaaataagccgggtgtgatggtgcacgcctgtaatcccag atacttgtgaggctgggacagaagaatcgcttgaacccgggaggcgaaggttgcagtgagccgagatcatgccactgcattccagcctgagcgacagagtgagaccttgtctcagaaaaaaaaaaatagggtgatttttatataaaaacactctcatttgttcttgaattaattgccatattgaacagattaaactcctttaaaccattttttctatctacaaacataattagtattcccataagcattttaagctccctttaaaaatttaatatgctcgattttcttttttcttttatttatt tatgtagttttcccagacagggtcttactctatcacccagtctggagtacagtgacacaatcctcacttactgtaacctcaagctccttgggatgaagcagtcctcccaccctagcctcttaagtagctgagattccaggcatgagccacctcacccagctgattttctttttaaacacatctaatacctgacaggtcgtaattacgactgtgcttggccatatcatcctaacacctaaagctcacttgtaaactgaaaattaaatttgaagggtgaattaacttctagccaacattccaat atcattctcacatttaatgaaattatcctacaactttgcttaaaagcataaaactagcatgtcagagtctcttaaaaattacaatcactattttaaataacaaacatacaggttaccctgaacttaacacctggttttaacatgatgaagttggtttcttttgtcattcttatactaaattctaaatcttcctggcacttaaggatatttacccaaggaagggggtgagatttaccggctcaggtacaccgaggttactggcttgaaaattcaagaccacagcatggtaaatttttcttcag agattcaaggttcatacataggacttgaagggcacatgctactcaaatgagtatatccaacttgagtgctacatcaacagggaccccatgtccactcttttcagtcgaaattggcttactatctacttcttctgggctattcaggttccgacaattgattgctttcagttaatttctttattgcccagttgaattctgcattcttctagattctttttgttccttcagcctcaatcataataccatttaatgacagtctcatatcctattagactagggtttctcagccttggcactattgacatatt gggtcagataattctttgtcctgtgcattgtagaatttagcatcttccttagcttctactcactagaagccagtagcactgtgtgcacatcctccttgccccctgccagctatgacaaccaaaatgtctccagacattaccaaatgtccactgagaggcaaattcactccgagttgacaacccctgtattaaacccaggggccctgcacttcttacatacttttgtctggtcaccagttgaaagtccttcttcttttttttttctctttttgagatggagtctcgctctgtcaccaggctggagtg cagtggcatgatctcggctcactgcagcctccacctcccgggttcaagcaattctcctgcctcagcctcctgagtagctgggattacaggcacgtgccaccacccagctaatttttgtatttttagtagaggtggggtttcatcatggtggccaggatggtctcaatctcttgacctcgtgatccgcccacctcggcctcccaaagtgctgggattacaggtgtgagccaccgtgcatgaccaaaagtccttctaatagcaaagtggaaaggtaggctgtgaacaatcttgaatgccata ctaagatttgtacttattctcataggcagtgaagagccatcaatagtttttacagaggctgacctgataactctggcggttcaatgtgcatgctatatactatatgcacaacacattaggaaggaagggaacagcgctcagagaaatcaaatcacttgcccatattacatagctcgtcagtggtggagtcagggtataatcagaaactcatgctccttcctctacctctccccagcacagagttggatcagcactgttcacccagctggtatccccatttcatggtaaagcctggaagc ttaggtctgactgatgcttgttgaaatacagactgtgggagcagactgcaggacagcattgtgacagagggtggggccaggagagagatgcggtaggaagactgttaagatgcatctcttattttctaggtggggcagctgcagctctcccacaatctgagtttggtgatcctggtaccccagaacctgaaacatcgtcttgaagacatggaacaggctctcagcccttctgttttcaaggccatcatggagaaactggagatgtccaagttccagcccactctcctaacactaccc cgcatcaaagtgacgaccagccaggatatgctctcaatcatggagaaattgggtgagctctggcagcttagggttactcccaggccatcagaggagaaaggggggatccctaagatgtagttagcattctctagagtattttttacatccataatctcagtttgtcctgcaaccctgcaagttagaggggtaggtgctattatcccattggatcattgtggaaatggaggctcagaagctttatgtgacttacccagagattccatgatttacccttgagctagtcagaggcaaacca gaacaccgacccaggtctccagctctcctgagttttttctatggttccttgtgacataggcagtggaacagtgggacaggtaaccgaggtgaattatggatgctccttccccagacacatttcaaaacagtcagccaccccgtggaatatgcaagaagctatctggaagtgcagatctggaggcttctgagtttacgagaggcaacagagactccattttcttttttttttttttttttgagacggagtcttgctctgtcacccaggacaggctggagtgcagtggtgcgatctctgctcac tgcaagatccgcctcccagattcacgccatgccattctctcgcctcagcctcccgagtagctgggactacaggtgcccgccaccacgcctggctaattttgtttttgtatttttagtagagacggggtttcactgtgttaaccaggatggtcacaatctccggaccttgtgatccacctgcctcggcctcccaaagtgctgggattacaggcatgagtcaccgcgcccagcccagagactccattttctaacctttgtttttttgtttgtttgtttttgagacggagcctcactctgtcacccag gctggagcgcagtggcatgatcttgggtcactgcaacctccgcctcctgggttcaagtgattctcctacctcagcctcccaagtagctgggattacaggcacctaccaccacgcccagctaatttttttgtatttttagtagagatggggtttcaccatgttggtgaagctggtcttgaactcctgggctcaagtgatccacccacctcggcttcccaaagtgctgggattacaggtgtgagccactgtgcccagcctcattttctaacctttgatctcatgtccagccctgtcacttcatttct tgttagagaatttgaccgcctaacacatgattccatttccttgtatatgtcatctgtataggaagaaaacctcttttttattttcattcaacacttctggccaccaaacaggtgtgggagtgttttctaagcaattctccagttatctggggacaccaactgggtgtcctacaattgaatccaattctaacactctacctggaaataacatcagatcccgtgcagtttaagtgctccgccccagcaaatttccccccgtttccaatgtcagttacaagactgggttgtcacctgtgcatcagac DIDDE HASLUND, KRISTIAN SKIPPER & ANNE caaccagctataatttggagattcccacaactccctgctagggtttcatcatttgctagaatagctgacaaaactcaggagacacttacatttactggtttattatgtaaaggattaagtaaaggatacagatgaacagccagttgaagagatacatagggcaaaatctggaagggtcctgggcccagcagcatctatcctgtggagttctgtttgctaccttcctgacacatgtatgtgttcaccaaccagaaactctcagaaccctgtactttaggagaagtcatcatgtaggcat KRUSE HOLLENSEN 3. FEBRUAR 2015 gattgattattacccaatctccagctcctctcttctccccagaggatggagagaggggctgaaagttccaagcttctaatcacacttggtctttctggtgaccagccccaatccaggagccagcaggagtagcctcattagcacaaaaggcattcctatgacccaggaaattccaagggatctagtagctctgtgtcaggaactgggatcggagaccaaatattagatggtctaatatttggagctaaagatgctcctatagcatattttcccattatttcacagcatcatggcattgc atataacctcattttttcatatatgacctatctctgatctattacctcacttccaaggtcctgggagataaggaggcaatgacatcatttttcagggttgttttagggatgaggggcaaggcctctcaccttgagagagagtggcagtgttgggcccactaatagaggatcccacgaactgccagagggtacagtatgtaatctggcaaacaagggaagaggaagagagcactgggactcaggatgaacccagagaattcaggacaaaggtctccatcagctgagggtatcatgct ggcttctgactctgtttttctctggttttgccctagaattcttcgatttttcttatgaccttaacctgtgtgggctgacagaggacccagatcttcaggtttctgcgatgcagcaccagacagtgctggaactgacagagactggggtggaggcggctgcagcctccgccatctctgtggcccgcaccctgctggtctttgaagtgcagcagcccttcctcttcgtgctctgggaccagcagcacaagttccctgtcttcatggggcgagtatatgaccccagggcctgagacctgcaggatc aggttagggcgagcgctacctctccagcctcagctctcagttgcagccctgctgctgcctgcctggacttggcccctgccacctcctgcctcaggtgtccgctatccaccaaaagggctccctgagggtctgggcaagggacctgcttctattagcccttctccatggccctgccatgctctccaaaccactttttgcagctttctctagttcaagttcaccagactctataaataaaacctgacagaccatgactttc AU AARHUS UNIVERSITET HVAD MED HAE? ctctgccctttgttgcaagggctggggagaacacc ctctgccctttgttgcaggggctggggagaacacc AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 HVAD MED SCID? Forbedret SCID genterapi Guider det raske gen ind i en ’sikker havn’ Mindsker risikoen for leukæmi AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 HVAD SKAL VI BEHANDLE? Type 2 diabetes HIV Hæmofili (blødersygdom) Skoldkopper SCID AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 EKSTRA LÆSNING TED talks: Gene therapy – the time is now: Nick Leschly Hope – It’s in our Genes: Dr. Barry Byrne Gene therapy for the treatment of Hemophilia B: Andrew M. Davidoff, MD www.giehmlab.dk http://haescan.org/ www.esgct.eu www.etiskraad.dk AU AARHUS UNIVERSITET DIDDE HASLUND, KRISTIAN SKIPPER & ANNE KRUSE HOLLENSEN 3. FEBRUAR 2015 AU AARHUS UNIVERSITET
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