J
Jenny McIntosh
Researcher at University College London
Publications - 62
Citations - 5490
Jenny McIntosh is an academic researcher from University College London. The author has contributed to research in topics: Genetic enhancement & Viral vector. The author has an hindex of 23, co-authored 59 publications receiving 4846 citations. Previous affiliations of Jenny McIntosh include University of Ulm & National Blood Service.
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Journal ArticleDOI
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
Amit C. Nathwani,Edward G. D. Tuddenham,Savita Rangarajan,Cecilia Rosales,Jenny McIntosh,David C. Linch,Pratima Chowdary,Anne Riddell,Arnulfo Jaquilmac Pie,Chris Harrington,James O'Beirne,Keith Smith,John Pasi,Bertil Glader,Pradip Rustagi,Catherine Y.C. Ng,Mark A. Kay,Junfang Zhou,Yunyu Spence,Christopher L. Morton,James A. Allay,John Coleman,Susan Sleep,John M. Cunningham,Deokumar Srivastava,Etiena Basner-Tschakarjan,Federico Mingozzi,Katherine A. High,John T. Gray,Ulrike M. Reiss,Arthur W. Nienhuis,Andrew M. Davidoff +31 more
TL;DR: Peripheral-vein infusion of scAAV2/8-LP1-hFIXco resulted in FIX transgene expression at levels sufficient to improve the bleeding phenotype, with few side effects.
Journal ArticleDOI
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
Amit C. Nathwani,Ulreke M Reiss,Edward G. D. Tuddenham,Cecilia Rosales,Cecilia Rosales,Pratima Chowdary,Jenny McIntosh,Marco Della Peruta,Elsa Lheriteau,Nishal Patel,Deepak Raj,Deepak Raj,Anne Riddell,Jun Pie,Savita Rangarajan,Savita Rangarajan,David H. Bevan,Michael Recht,Yu-min P Shen,Kathleen Halka,Etiena Basner-Tschakarjan,Federico Mingozzi,Katherine A. High,James A. Allay,Mark A. Kay,Catherine Y.C. Ng,Junfang Zhou,Maria I Cancio,Christopher L. Morton,John T. Gray,Deo Kumar Srivastava,Arthur W. Nienhuis,Andrew M. Davidoff +32 more
TL;DR: In 10 patients with severe hemophilia B, the infusion of a single dose of AAV8 vector resulted in long-term therapeutic factor IX expression associated with clinical improvement, and with a follow-up period of up to 3 years, no late toxic effects from the therapy were reported.
Journal ArticleDOI
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
Amit C. Nathwani,John T. Gray,Catherine Y.C. Ng,Junfang Zhou,Yunyu Spence,Simon N. Waddington,Edward G. D. Tuddenham,Geoffrey Kemball-Cook,Jenny McIntosh,Mariëtte Boon-Spijker,Koen Mertens,Andrew M. Davidoff +11 more
TL;DR: A liver-restricted mini-human factor IX (hFIX) expression cassette is constructed that can be packaged as complementary dimers within individual AAV particles that resulted in a 20-fold improvement in hFIX expression in mice over comparable ssAAV vectors.
Journal ArticleDOI
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.
Amit C. Nathwani,Amit C. Nathwani,Cecilia Rosales,Jenny McIntosh,Ghasem Rastegarlari,Devhrut Nathwani,Deepak Raj,Sushmita Nawathe,Simon N. Waddington,Roderick T. Bronson,Scott Jackson,Robert E. Donahue,Katherine A. High,Federico Mingozzi,Catherine Y.C. Ng,Junfang Zhou,Yunyu Spence,M. Beth McCarville,Marc Valentine,James A. Allay,John Coleman,Susan Sleep,John T. Gray,Arthur W. Nienhuis,Andrew M. Davidoff +24 more
TL;DR: The long-term consequences of a single intravenous administration of a self-complementary AAV vector encoding a codon optimized human factor IX gene in 24 nonhuman primates revealed no toxicity, and data support further evaluation of this vector in hemophilia B patients.
Journal ArticleDOI
Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
Amit C. Nathwani,John T. Gray,Jenny McIntosh,Catherine Y.C. Ng,Junfang Zhou,Yunyu Spence,Melanie Cochrane,Elaine Gray,Edward G. D. Tuddenham,Andrew M. Davidoff +9 more
TL;DR: The safety and efficacy of peripheral venous administration of a self-complementary adeno-associated vector encoding the human FIX gene (scAAV-LP1-hFIXco) was evaluated in nonhuman primates for gene therapy of hemophilia B.