Scienceisawayofthinkingmuchmorethanitisabodyofknowledge.

CarlSagan

Isabella Saggio

www.saggiolab.com

GeneTherapy

Ridiculous idea

Cures all known diseases

Woudn’t give it to my dog

Good treatment

Modifief from E.WFW. Alton, London ESGT Meeting 99

1999Gelsinger 2010

1990

2003 tumor cases

De Silva

2008 Mohr case

2002

http://www.paolobiancolab.it/science-society-1/

Course:GTTexts•  AdvancedTextbookonGeneTransfer,GeneTherapyandGene7cPharmacologyWorld

Scien7ficScherman2014•  Stemcell:biologyandengineering/PucVanPham.Springer.2018ebook•  GeneTherapyinNeurologicalDisorders1stEdi7on,KindleEdi,on•  CellandGeneTherapies(AdvancesandControversiesinHematopoie7cTransplanta7onand

CellTherapy)1sted.2019Edi7on,KindleEdi,on

•  Geneandcelltherapy:therapeu>cmechanismsandstrategies/editedbyNancySmythTempleton

•  Thedevelopmentofhumangenetherapy/editedbyTheodoreFriedmann•  Genetherapy:principlesandapplica>ons/editedbyThomasBlankenstein•  Cancergenetherapy/editedbyDavidT.Curiel,JoanneT.Douglas•  Conceptsingene>cmedicine/ed.byBoroDropulic,BarrieCarterwww.saggiolab.com;psw2013/2014Exam:Posterpresenta,onforpeoplea7endingWri7enoronlineexamforpeoplenota7ending

Gene Therapy�=�

deliberate transfer of DNA for therapeutic purposes

Subjects:Group I: Telomeropathies (including laminopathies) Group II: CancerGroup III: Fibrous dysplasia

Phase 1: background Phase 2: project presentationSignature for groups

Prepare poster , in the notes insert the text

Project

Enabling technology (I)•  1928 Transformation with pneumococcus•  1944 DNA is a transforming substance of pn.•  1952 DNA is genetic material (phage)•  1953 Double helix structure•  1963 Genetic code elucidated•  1968 Transformation by DNA viruses

Enabling technology (II)

•  1976 Cloning of globin genes•  1979 Cloned gene expressed•  1981 Clinical thalassemia studies•  1982 Recombinant proteins for clinical use•  1985 Retroviral vectors developed•  1990 First ADA trials•  1991 CF protocols•  1992 Large number of cancer trials•  1998 Rna interference-gene therapy of dominant

genetic diseases

Clinical trials – 2016 update

Trials/continent

Trials/country

3yrs ago

• PhaseI:smallnumberofpa>ents(20-80)fprpharmacovigilance,pharmacokine>cs,pharmacodynamics

• PhaseII:performedonlargergroups(20-300)anddesignedtoassessclinicalefficacyofthetherapy;aswellastocon>nuePhaseIassessments

• PhaseIII:randomizedcontrolledtrialsonlargepa>entgroups(300-3000ormoredependinguponthecondi>on)andareaimedatbeingthedefini>veassessmentoftheefficacyofthenewtherapy,incomparisonwithcurrent'GoldStandard'treatment.

• PhaseIV:involvethepost-launchsafetysurveillanceandongoingtechnicalsupportofadrug.

Which are the trial phases

Gene therapy trial phases

Which diseases with gene therapy

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