Arthur W. Nienhuis - Publications, H-index & Citations

Arthur W. Nienhuis

Children's Hospital of Philadelphia

81H指数

347论文数

2.3W被引数

收录论文 28

发表时间

High level of fetal-globin reactivation by designed transcriptional activator-like effector

BLOOD ADVANCES

IF7.1

2020-02-21

OAAI

Zhan, Jun; Irudayam, Maria Johnson; Nakamura, Yukio; Kurita, Ryo; Nienhuis, Arthur W.

George Stamatoyannopoulos (1934-2018) Obituary

MOLECULAR THERAPY

IF12

2018-08-01

OAAI

Dunbar, Cynthia E.; Bodine, David M.; Nienhuis, Arthur W.

Gene Therapy for Hemophilia

MOLECULAR THERAPY

IF12

2017-05-01

OAAI

Nienhuis, Arthur W.; Nathwani, Amit C.; Davidoff, Andrew M.

Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia

MOLECULAR THERAPY

IF12

2017-03-01

OAAI

Zhao, Hui Fen; Abraham, Allistair; Kim, Yoon-Sang; Wang, Yong-Dong; Pestina, Tamara; Zhan, Jun; Humphries, Keith; Nienhuis, Arthur W.; Persons, Derek A.

Lentiviral Hematopoietic Stem Cell Gene Therapy for Sjogren-Larsson Syndrome

MOLECULAR THERAPY

IF12

2016-05-01

OAAI

Kim, Yoon-Sang; Johnson, Irudayam Maria; S'Aulis, Dana; Rizzo, William B.; Nienhuis, Arthur W.

Forced Fetal-Globin Reactivation Mediated by the TALE-ldb1 Fusion Protein

MOLECULAR THERAPY

IF12

2016-05-01

OAAI

Zhan, Jun; Johnson, Irudayam Maria; Nienhuis, Arthur

Lentiviral Vector Particles Pseudotyped with Wild-Type Baboon Endogenous Retrovirus (BAEV) Glycoprotein Outperform VSV-G Particles in Transducing Human CD34+Cells Isolated from Cytokine-Mobilized Peripheral Blood or Bone Marrow

MOLECULAR THERAPY

IF12

2016-05-01

OAAI

Hargrove, Phillip; Kim, Yoon-Sang; Wielgosz, Matthew; Nienhuis, Arthur W.

Distribution of AAV8 Particles in Cell Lysates and Supernatants Changes with Time and Is Dependent on the Vector Genome

MOLECULAR THERAPY

IF12

2015-05-01

OAAI

Piras, Bryan A.; Lockey, Timothy D.; Davidoff, Andrew; Nienhuis, Arthur; d'Azzo, Alessandra; Nathwani, Amit; Meagher, Michael

Safe and Effective Gene Therapy for Wiskott - Aldrich Syndrome Using an Insulated Lentiviral Vector

MOLECULAR THERAPY

IF12

2015-05-01

OAAI

Singh, Swati; Sather, Blythe; Khan, Iram; Kim, Socheath; Seymour, Brenda; Wielgosz, Matthew; Kiem, Hans-Peter; Adair, Jennifer; Liggett, Denny; Nienhuis, Arthur; Rawlings, David J.

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

NEW ENGLAND JOURNAL OF MEDICINE

IF78.5

2014-11-20

1.1K

OAAI

Nathwani, A. C.; Reiss, U. M.; Tuddenham, E. G. D.; Rosales, C.; Chowdary, P.; McIntosh, J.; Della Peruta, M.; Lheriteau, E.; Patel, N.; Raj, D.; Riddell, A.; Pie, J.; Rangarajan, S.; Bevan, D.; Recht, M.; Shen, Y. -M.; Halka, K. G.; Basner-Tschakarjan, E.; Mingozzi, F.; High, K. A.; Allay, J.; Kay, M. A.; Ng, C. Y. C.; Zhou, J.; Cancio, M.; Morton, C. L.; Gray, J. T.; Srivastava, D.; Nienhuis, A. W.; Davidoff, A. M.

Preclinical Dose-Finding Study With a Liver-Tropic, Recombinant AAV-2/8 Vector in the Mouse Model of Galactosialidosis

MOLECULAR THERAPY

IF12

2012-02-01

OAAI

Hu, Huimin; Gomero, Elida; Bonten, Erik; Gray, John T.; Allay, Jim; Wu, Yanan; Wu, Jianrong; Calabrese, Christopher; Nienhuis, Arthur; d'Azzo, Alessandra

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

NEW ENGLAND JOURNAL OF MEDICINE

IF78.5

2011-12-22

1.7K

OAAI

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.

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

MOLECULAR THERAPY

IF12

2011-05-01

319

OAAI

Nathwani, Amit C.; Rosales, Cecilia; McIntosh, Jenny; Rastegarlari, Ghasem; Nathwani, Devhrut; Raj, Deepak; Nawathe, Sushmita; Waddington, Simon N.; Bronson, Roderick; Jackson, Scott; Donahue, Robert E.; High, Katherine A.; Mingozzi, Federico; Ng, Catherine Y. C.; Zhou, Junfang; Spence, Yunyu; McCarville, M. Beth; Valentine, Marc; Allay, James; Coleman, John; Sleep, Susan; Gray, John T.; Nienhuis, Arthur W.; Davidoff, Andrew M.

Transduction of Human Primitive Repopulating Hematopoietic Cells With Lentiviral Vectors Pseudotyped With Various Envelope Proteins

MOLECULAR THERAPY

IF12

2010-07-01

OAAI

Kim, Yoon-Sang; Wielgosz, Matthew M.; Hargrove, Phillip; Kepes, Steven; Gray, John; Persons, Derek A.; Nienhuis, Arthur W.

Dose response results of self complementary adeno-associated virus (AAV) vector-mediated factor IX gene transfer in non-human primates

HAEMOPHILIA

IF3

2009-03-23

PREAI

Reiss, Ulrike; Davidoff, Andrew; Gray, John; Nienhuis, Arthur; Nathwani, Amit