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Funded by the Medical Research Council and others; ClinicalTrials.gov number, NCT00979238.

Analysis of institutional authors

Rosales, CeciliaAuthor

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November 13, 2024
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Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B

Publicated to:New England Journal Of Medicine. 365 (25): 2357-2365 - 2011-12-22 365(25), DOI: 10.1056/NEJMoa1108046

Authors: Nathwani, Amit C; Tuddenham, Edward G D; Rangarajan, Savita; Rosales, Cecilia; McIntosh, Jenny; Linch, David C; Chowdary, Pratima; Riddell, Anne; Pie, Arnulfo Jaquilmac; Harrington, Chris; O'Beirne, James; Smith, Keith; Pasi, John; Glader, Bertil; Rustagi, Pradip; Ng, Catherine Y C; Kay, Mark A; Zhou, Junfang; Spence, Yunyu; Morton, Christopher L; Allay, James; Coleman, John; Sleep, Susan; Cunningham, John M; Srivastava, Deokumar; Basner-Tschakarjan, Etiena; Mingozzi, Federico; High, Katherine A; Gray, John T; Reiss, Ulrike M; Nienhuis, Arthur W; Davidoff, Andrew M

Affiliations

Barts & London Queen Marys Sch Med & Dent, Ctr Haematol, London, England - Author
Basingstoke & N Hampshire NHS Fdn Trust, Basingstoke, Hants, England - Author
Childrens GMP, Memphis, TN USA - Author
Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA - Author
Howard Hughes Med Inst, Chevy Chase, MD USA - Author
NHS Blood & Transplant, London, England - Author
Royal Free Natl Hlth Serv NHS Trust, Katharine Dormandy Haemophilia Ctr, London, England - Author
Royal Free Natl Hlth Serv NHS Trust, Liver Unit, London, England - Author
Royal Free Natl Hlth Serv NHS Trust, Thrombosis Unit, London, England - Author
St Jude Childrens Hosp, Dept Biostat, Memphis, TN 38105 USA - Author
St Jude Childrens Hosp, Dept Hematol, Memphis, TN 38105 USA - Author
St Jude Childrens Hosp, Dept Surg, Memphis, TN 38105 USA - Author
Stanford Univ, Sch Med, Palo Alto, CA 94304 USA - Author
UCL, Inst Canc, Dept Haematol, London WC1E 6BT, England - Author
Univ Chicago, Dept Pediat, Chicago, IL 60637 USA - Author
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Abstract

BACKGROUND 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. METHODS We infused a single dose of a serotype-8-pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing a codon-optimized human factor IX (FIX) transgene (scAAV2/8-LP1-hFIXco) in a peripheral vein in six patients with severe hemophilia B (FIX activity,

Keywords

Adenoassociated viral vectorAdultClinical-trialDependovirusEfficient transductionExercisExpressionFactor ixGenetic therapyGenetic vectorsHemophilia bHuman fixHuman-factor ixHumansImmune-responseInfusions, intravenousLiverMiddle agedTherapyTransgenes

Quality index

Bibliometric impact. Analysis of the contribution and dissemination channel

The work has been published in the journal New England Journal Of Medicine due to its progression and the good impact it has achieved in recent years, according to the agency WoS (JCR), it has become a reference in its field. In the year of publication of the work, 2011, it was in position 1/155, thus managing to position itself as a Q1 (Primer Cuartil), in the category Medicine, General & Internal.

From a relative perspective, and based on the normalized impact indicator calculated from the Field Citation Ratio (FCR) of the Dimensions source, it yields a value of: 213.4, which indicates that, compared to works in the same discipline and in the same year of publication, it ranks as a work cited above average. (source consulted: Dimensions Aug 2025)

Specifically, and according to different indexing agencies, this work has accumulated citations as of 2025-08-02, the following number of citations:

  • WoS: 1439
  • Europe PMC: 1114

Impact and social visibility

From the perspective of influence or social adoption, and based on metrics associated with mentions and interactions provided by agencies specializing in calculating the so-called "Alternative or Social Metrics," we can highlight as of 2025-08-02:

  • The use, from an academic perspective evidenced by the Altmetric agency indicator referring to aggregations made by the personal bibliographic manager Mendeley, gives us a total of: 1033.
  • The use of this contribution in bookmarks, code forks, additions to favorite lists for recurrent reading, as well as general views, indicates that someone is using the publication as a basis for their current work. This may be a notable indicator of future more formal and academic citations. This claim is supported by the result of the "Capture" indicator, which yields a total of: 1060 (PlumX).

With a more dissemination-oriented intent and targeting more general audiences, we can observe other more global scores such as:

  • The Total Score from Altmetric: 364.55.
  • The number of mentions on the social network Facebook: 10 (Altmetric).
  • The number of mentions on the social network X (formerly Twitter): 56 (Altmetric).
  • The number of mentions on Wikipedia: 2 (Altmetric).
  • The number of mentions in news outlets: 29 (Altmetric).

Leadership analysis of institutional authors

This work has been carried out with international collaboration, specifically with researchers from: United Kingdom; United States of America.