Xavier M Anguela

Affiliations

Spark Therapeutics, Inc., Philadelphia, Pennsylvania 19104, USA; email: xavier.anguela@sparktx.com , kathy.high@sparktx.com.

Publications (8)

Entering the Modern Era of Gene Therapy.

Annual review of medicine

Avec: Xavier M Anguela , Katherine A High

Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice.

Molecular therapy. Methods & clinical development

Avec: Chenhui Zou , Koen O A Vercauteren , Eleftherios Michailidis , Mohammad Kabbani , Irene Zoluthkin , Corrine Quirk , Luis Chiriboga , Mustafa Yazicioglu , Xavier M Anguela , Philip Meuleman , Katherine A High , Roland W Herzog , Ype P de Jong

Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A.

Molecular therapy. Methods & clinical development

Avec: Liron Elkouby , Sean M Armour , Raffaella Toso , Marti DiPietro , Robert J Davidson , Giang N Nguyen , Mallory Willet , Stephanie Kutza , Joseph Silverberg , Jennifer Frick , Marco Crosariol , Yuhuan Wang , Chuansong Wang , Katherine A High , Denise E Sabatino , Xavier M Anguela

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.

The New England journal of medicine

Avec: Lindsey A George , Paul E Monahan , M Elaine Eyster , Spencer K Sullivan , Margaret V Ragni , Stacy E Croteau , John E J Rasko , Michael Recht , Benjamin J Samelson-Jones , Amy MacDougall , Kristen Jaworski , Robert Noble , Marla Curran , Klaudia Kuranda , Federico Mingozzi , Tiffany Chang , Kathleen Z Reape , Xavier M Anguela , Katherine A High

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies.

Nature medicine

Avec: Christian Leborgne , Elena Barbon , Jeffrey M Alexander , Hayley Hanby , Sandrine Delignat , Daniel M Cohen , Fanny Collaud , Saghana Muraleetharan , Dan Lupo , Joseph Silverberg , Karen Huang , Laetitia van Wittengerghe , Béatrice Marolleau , Adeline Miranda , Anna Fabiano , Victoria Daventure , Heena Beck , Xavier M Anguela , Giuseppe Ronzitti , Sean M Armour , Sebastien Lacroix-Desmazes , Federico Mingozzi

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.

Molecular therapy : the journal of the American Society of Gene Therapy

Avec: Lindsey A George , Margaret V Ragni , John E J Rasko , Leslie J Raffini , Benjamin J Samelson-Jones , Margareth Ozelo , Maria Hazbon , Alexa R Runowski , Jennifer A Wellman , Katie Wachtel , Yifeng Chen , Xavier M Anguela , Klaudia Kuranda , Federico Mingozzi , Katherine A High

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

Nature communications

Avec: Helena Costa-Verdera , Fanny Collaud , Christopher R Riling , Pauline Sellier , Jayme M L Nordin , G Michael Preston , Umut Cagin , Julien Fabregue , Simon Barral , Maryse Moya-Nilges , Jacomina Krijnse-Locker , Laetitia van Wittenberghe , Natalie Daniele , Bernard Gjata , Jeremie Cosette , Catalina Abad , Marcelo Simon-Sola , Severine Charles , Mathew Li , Marco Crosariol , Tom Antrilli , William J Quinn , David A Gross , Olivier Boyer , Xavier M Anguela , Sean M Armour , Pasqualina Colella , Giuseppe Ronzitti , Federico Mingozzi

Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec.

Blood advances

Avec: Xavier M Anguela , Katherine A High

Réseau de co-auteurs

Katherine A High 6 collaborations

Federico Mingozzi 4 collaborations

Sean M Armour 3 collaborations

Joseph Silverberg 2 collaborations

Marco Crosariol 2 collaborations

Lindsey A George 2 collaborations

Margaret V Ragni 2 collaborations

John E J Rasko 2 collaborations

Benjamin J Samelson-Jones 2 collaborations

Klaudia Kuranda 2 collaborations

Fanny Collaud 2 collaborations

Giuseppe Ronzitti 2 collaborations

Chenhui Zou 1 collaboration

Koen O A Vercauteren 1 collaboration

Eleftherios Michailidis 1 collaboration

Mohammad Kabbani 1 collaboration

Irene Zoluthkin 1 collaboration

Corrine Quirk 1 collaboration

Luis Chiriboga 1 collaboration

Mustafa Yazicioglu 1 collaboration

Philip Meuleman 1 collaboration

Roland W Herzog 1 collaboration

Ype P de Jong 1 collaboration

Liron Elkouby 1 collaboration

Raffaella Toso 1 collaboration

Marti DiPietro 1 collaboration

Robert J Davidson 1 collaboration

Giang N Nguyen 1 collaboration

Mallory Willet 1 collaboration

Stephanie Kutza 1 collaboration

Jennifer Frick 1 collaboration

Yuhuan Wang 1 collaboration

Chuansong Wang 1 collaboration

Denise E Sabatino 1 collaboration

Paul E Monahan 1 collaboration

M Elaine Eyster 1 collaboration

Spencer K Sullivan 1 collaboration

Stacy E Croteau 1 collaboration

Michael Recht 1 collaboration

Amy MacDougall 1 collaboration

Kristen Jaworski 1 collaboration

Robert Noble 1 collaboration

Marla Curran 1 collaboration

Tiffany Chang 1 collaboration

Kathleen Z Reape 1 collaboration

Christian Leborgne 1 collaboration

Elena Barbon 1 collaboration

Jeffrey M Alexander 1 collaboration

Hayley Hanby 1 collaboration

Sandrine Delignat 1 collaboration

Daniel M Cohen 1 collaboration

Saghana Muraleetharan 1 collaboration

Dan Lupo 1 collaboration

Karen Huang 1 collaboration

Laetitia van Wittengerghe 1 collaboration

Béatrice Marolleau 1 collaboration

Adeline Miranda 1 collaboration

Anna Fabiano 1 collaboration

Victoria Daventure 1 collaboration

Heena Beck 1 collaboration

Sebastien Lacroix-Desmazes 1 collaboration

Leslie J Raffini 1 collaboration

Margareth Ozelo 1 collaboration

Maria Hazbon 1 collaboration

Alexa R Runowski 1 collaboration

Jennifer A Wellman 1 collaboration

Katie Wachtel 1 collaboration

Yifeng Chen 1 collaboration

Helena Costa-Verdera 1 collaboration

Christopher R Riling 1 collaboration

Pauline Sellier 1 collaboration

Jayme M L Nordin 1 collaboration

G Michael Preston 1 collaboration

Umut Cagin 1 collaboration

Julien Fabregue 1 collaboration

Simon Barral 1 collaboration

Maryse Moya-Nilges 1 collaboration

Jacomina Krijnse-Locker 1 collaboration

Laetitia van Wittenberghe 1 collaboration

Natalie Daniele 1 collaboration

Bernard Gjata 1 collaboration

Jeremie Cosette 1 collaboration

Catalina Abad 1 collaboration

Marcelo Simon-Sola 1 collaboration

Severine Charles 1 collaboration

Mathew Li 1 collaboration

Tom Antrilli 1 collaboration

William J Quinn 1 collaboration

David A Gross 1 collaboration

Olivier Boyer 1 collaboration

Pasqualina Colella 1 collaboration