Arthur W. Nienhuis
St. Jude Children's Research Hospital
332 Papers
7.4K Citations
Arthur W. Nienhuis is an academic researcher from St. Jude Children's Research Hospital. The author has contributed to research in topics: Gene & Globin. The author has an hindex of 85, co-authored 332 publications. Previous affiliations of Arthur W. Nienhuis include National Institutes of Health & Walsh University.
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Papers
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.
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.
Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major.
Gary M. Brittenham,Patricia Griffith,Arthur W. Nienhuis,Christine E. McLaren,Neal S. Young,Eben E. Tucker,Christopher J. Allen,David E. Farrell,John W. Harris +8 more
TL;DR: The early use of deferoxamine in an amount proportional to the transfusional iron load reduces the body iron burden and helps protect against diabetes mellitus, cardiac disease, and early death in patients with thalassemia major.
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An oligomer complementary to c-myc mRNA inhibits proliferation of HL-60 promyelocytic cells and induces differentiation
TL;DR: The effects of the antisense oligomer exhibited sequence specificity, and furthermore, these effects could be reversed by hybridization competition with another complementary oligomer.
529
Inducible production of c-fos antisense RNA inhibits 3T3 cell proliferation
TL;DR: Results indicate that the strategy of generating antisense RNA to inhibit gene expression may be useful in delineating the function of protooncogenes.
498