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Nature Medicine, ISSN 1078-8956, 12/2018, Volume 24, Issue 12, pp. 1899 - 1909
Journal Article
Journal Article
Journal of translational medicine, ISSN 1479-5876, 2012, Volume 10, Issue 1, pp. 122 - 122
Background: Adeno-associated vectors (rAAV) have been used to attain long-term liver gene expression. In humans, the cellular immune response poses a serious... 
MEDICINE, RESEARCH & EXPERIMENTAL | Neutralizing antibodies | Immunomodulation | ADENOASSOCIATED VIRUS | Re-administration | TRANSDUCTION | ADENOVIRUS | VECTOR | REPLICATION | IMMUNE-RESPONSES | Vector Immunology/Host Responses | EXPRESSION | Adeno-associated virus serotype 5 | MYCOPHENOLIC-ACID | Macaca fascicularis - immunology | Injections, Intravenous | Genetic Vectors - administration & dosage | Immunity, Humoral - immunology | Humans | Dependovirus - drug effects | Immunity - drug effects | Liver - drug effects | Mycophenolic Acid - pharmacology | Treatment Failure | Female | Immunosuppressive Agents - pharmacology | Immunosuppressive Agents - administration & dosage | Serotyping | Gene Transfer Techniques | Dependovirus - metabolism | Liver - metabolism | Transgenes - genetics | Mice, Transgenic | Genetic Vectors - genetics | DNA, Viral - blood | Antigens, Viral - immunology | Capsid - immunology | Immunosuppression | Animals | Mycophenolic Acid - analogs & derivatives | Immunity, Humoral - drug effects | Mice | Immune response | Corticosteroids | Liver | Genes | Genomics | Fluorescence | Dependoviruses | T cells | Gene expression | Immunosuppressive agents | Metabolites | Analysis | Immunotherapy | Primates | Genetic research | Gene therapy | Proteins | Polymerase chain reaction | Rodents | Hepatology | Experiments | Manuscripts | Deoxyribonucleic acid--DNA | Immune system
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 2019
Introduction: Acute intermittent porphyria (AIP) is characterized by hepatic over-production of the heme precursors when aminolevulinic acid (ALA)-synthase 1... 
Porphyrin precursor excretion | Acute intermittent porphyria, in silico analysis | Mechanistic modelling
Journal Article
PLoS ONE, ISSN 1932-6203, 01/2014, Volume 9, Issue 1, p. e85432
Journal Article
by Schmidt, Manfred and Verhoeyen, Els and Gobbo, Emilie and Divers, Dominique and Oudrhiri, Noufissa and Griscelli, Frank and Bennaceur-Griscelli, Annelise and Klatzmann, David and Anguela, Xavier M and Sharma, Rajiv and Sharma, Rajiv and Li, Hojun and Li, Hojun and Haurigot, Virginia and Haurigot, Virginia and Haurigot, Virginia and Bhagwat, Anand and Bhagwat, Anand and Davidson, Robert and Davidson, Robert and Zhou, Shangzhen and Zhou, Shangzhen and Zhou, Shangzhen and Doyon, Yannick and Gregory, Philip D and Gregory, Philip D and Gregory, Philip D and Holmes, Michael C and Holmes, Michael C and Holmes, Michael C and High, Katherine A and Carbonaro, Denise and Shaw, Kit and Jin, Xiangyang and Geiger, Sabine and Mishra, Suparna and Cooper, Aaron and DeOliveira, Satiro and Sokolic, Rob and Candotti, Fabio and Carmo, Marlene and Arumugam, Paritha and Alonso-Ferrero, Maria and Schambach, Axel and Schambach, Axel and Baum, Christopher and Baum, Christopher and Baum, Christopher and Risma, Kimberly and Malik, Punam and Jordan, Michael and Rivat, Christine and Booth, Claire and Thrasher, Adrian and Whilding, Lynsey and Archibald, Kyra and Oberg, Daniel and Golan, Talia and Hubert, Ayala and Shemi, Amotz and Khvalevsky, Elina Zorde and Gabai-Malka, Racheli and Focht, Gili and Brunschwig, Zivia and Raskin, Stephen and Goldberg, Nahum and Ben-David, Eli and Peretz, Tamar and Eliakim, Rami and Dankur, Alan and Galun and Rachmur, Itzik and Domb, Avi and Kopelman, Yael and Hantz, Yael and Lahav, Mor and Arbel-Alon, Sagit and Dickson, George and Barkats, Martine and Daboussi, Fayza and Silva, Georges and Cedrone, Frederic and Epinat, Jean Charles and Juillerat, Alexandre and Valton, Julien and Montini, Eugenio and Biffi, Alessandra and Biffi, Alessandra and Calabria, Andrea and Calabria, Andrea and Biasco, Luca and Biasco, Luca and Cesani, Martina and Cesani, Martina and Benedicenti, Fabrizio and Benedicenti, Fabrizio and Plati, Tiziana and Leo, Simone and Zanetti, Gianluigi and Aiuti, Alessandro and ...
Human Gene Therapy, ISSN 1043-0342, 10/2012, Volume 23, Issue 10, pp. A1 - A173
Journal Article
Human Molecular Genetics, ISSN 0964-6906, 11/2018, Volume 27, Issue 21, pp. 3688 - 3696
Abstract A first-in-human gene therapy trial using a recombinant adeno-associated viral (rAAV) vector for acute intermittent porphyria (AIP) reveals that... 
NONHUMAN-PRIMATES | SYNTHASE | LIVER-TRANSPLANTATION | NEUROPATHY | BIOCHEMISTRY & MOLECULAR BIOLOGY | GENETICS & HEREDITY | PROTECTS
Journal Article
PLoS ONE, ISSN 1932-6203, 03/2012, Volume 7, Issue 3, p. e32978
Journal Article