Gene Therapy: Principles and Clinical PotentialPublished on Oct 31, 2019· DOI :10.1017/9781108564434.053Copy DOISuzanne M. K. Buckley26Estimated H-index: 26 , Anna L. David30Estimated H-index: 30 Request Full-textCiteAbstract
References115NewestFetal gene therapy for neurodegenerative disease of infants.Sep 1, 2018·Nature Medicine30.64#1Giulia Massaro (UCL: University College London)H-Index: 4#2Citra Nurfarah Zaini Mattar (NUS: National University of Singapore)H-Index: 13Last.Ahad A. Rahim (UCL: University College London)H-Index: 18view all 22 authors...27 CitationsSourceCiteSavePerinatal and long-term effects of maternal uterine artery adenoviral VEGF-A165 gene therapy in the growth-restricted guinea pig fetus.Aug 1, 2018·American Journal of Physiology-regulatory Integrative and Comparative Physiology3.18#1O. R. Vaughan (UCL: University College London)H-Index: 15#2C Rossi (UCL: University College London)H-Index: 3Last.Anna L. David (UCL: University College London)H-Index: 30view all 10 authors...4 CitationsSourceCiteSavePrenatal Correction of X-Linked Hypohidrotic Ectodermal DysplasiaApr 26, 2018·The New England Journal of Medicine70.67#1Holm SchneiderH-Index: 17#2Florian FaschingbauerH-Index: 14Last.Pascal SchneiderH-Index: 79view all 15 authors...20 CitationsSourceCiteSaveGene Therapy in Patients with Transfusion-Dependent β-ThalassemiaApr 19, 2018·The New England Journal of Medicine70.67#1Alexis A. Thompson (NU: Northwestern University)H-Index: 34#2Mark C. Walters (Children's Hospital Oakland Research Institute)H-Index: 36Last.Marina Cavazzana-Calvo (University of Paris)H-Index: 58view all 47 authors...100 CitationsSourceCiteSaveAAV5-Factor VIII Gene Transfer in Severe Hemophilia A.Dec 28, 2017·The New England Journal of Medicine70.67#1Savita RangarajanH-Index: 20#2L WalshH-Index: 1Last.K. J. PasiH-Index: 11view all 11 authors...125 CitationsSourceCiteSaveIn Vitro Human Placental Studies to Support Adenovirus-Mediated VEGF-DΔNΔC Maternal Gene Therapy for the Treatment of Severe Early-Onset Fetal Growth RestrictionDec 11, 2017·Human gene therapy. Clinical development2.78#1Michelle DesforgesH-Index: 9#2Alexandra RogueH-Index: 8Last.Paul BrownbillH-Index: 17view all 12 authors...3 CitationsSourceCiteSaveHemophilia B Gene Therapy with a High-Specific-Activity Factor IX VariantDec 7, 2017·The New England Journal of Medicine70.67#1Lindsey A. George (UPenn: University of Pennsylvania)H-Index: 8#2Spencer K. SullivanH-Index: 12Last.A HighKatherineH-Index: 73view all 28 authors...129 CitationsSourceCiteSaveScalable Lentiviral Vector Production Using Stable HEK293SF Producer Cell LinesDec 1, 2017·Human Gene Therapy Methods2.09#1P ManceurAzizaH-Index: 1#2KimHowardH-Index: 1Last.AnsorgeSvenH-Index: 1view all 12 authors...10 CitationsSourceCiteSaveLong-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophyDec 1, 2017·Nature Communications11.88#1Caroline Le Guiner (University of Nantes)H-Index: 17#2Laurent ServaisH-Index: 21Last.George Dickson (RHUL: Royal Holloway, University of London)H-Index: 47view all 35 authors...48 CitationsSourceCiteSaveSingle-Dose Gene-Replacement Therapy for Spinal Muscular AtrophyNov 2, 2017·The New England Journal of Medicine70.67#1Jerry R. MendellH-Index: 80#2Samiah Al-ZaidyH-Index: 8Last.Brian K. KasparH-Index: 40view all 26 authors...293 CitationsSourceCiteSave12345678910