Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B | Zendy

Amit C. Nathwani | Zendy; Edward G. D. Tuddenham | Zendy; Savita Rangarajan | Zendy; Cecilia Rosales | Zendy; Jenny McIntosh | Zendy; David C. Linch | Zendy; Pratima Chowdary | Zendy; Anne Riddell | Zendy; Arnulfo Pie | Zendy; Chris F. Harrington | Zendy; James O’Beirne | Zendy; Keith Smith | Zendy; John Pasi | Zendy; Bertil Glader | Zendy; Pradip K. Rustagi | Zendy; Catherine Y. Ng | Zendy; Mark A. Kay | Zendy; Junfang Zhou | Zendy; Yunyu Spence | Zendy; Christopher L. Morton | Zendy; James A. Allay | Zendy; John B. Coleman | Zendy; Susan Sleep | Zendy; John M. Cunningham | Zendy; Deo Kumar Srivastava | Zendy; Etiena BasnerTschakarjan | Zendy; Federico Mingozzi | Zendy; Katherine A. High | Zendy; John T. Gray | Zendy; Ulrike M. Reiss | Zendy; Arthur W. Nienhuis | Zendy; Andrew M. Davidoff | Zendy

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Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Author(s) -

Amit C. Nathwani,

Edward G. D. Tuddenham,

Savita Rangarajan,

Cecilia Rosales,

Jenny McIntosh,

David C. Linch,

Pratima Chowdary,

Anne Riddell,

Arnulfo Pie,

Chris F. Harrington,

James O’Beirne,

Keith Smith,

John Pasi,

Bertil Glader,

Pradip K. Rustagi,

Catherine Y. Ng,

Mark A. Kay,

Junfang Zhou,

Yunyu Spence,

Christopher L. Morton,

James A. Allay,

John B. Coleman,

Susan Sleep,

John M. Cunningham,

Deo Kumar Srivastava,

Etiena BasnerTschakarjan,

Federico Mingozzi,

Katherine A. High,

John T. Gray,

Ulrike M. Reiss,

Arthur W. Nienhuis,

Andrew M. Davidoff

Publication year - 2011

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1108046

Subject(s) - medicine , genetic enhancement , asymptomatic , factor ix , vector (molecular biology) , virus , viral vector , adeno associated virus , transgene , adenoviridae , virology , gastroenterology , immunology , gene , biology , recombinant dna , biochemistry

Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder.

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