Arthur W. Nienhuis

Biochemistry, Genetics and Molecular Biology · Life Sciences

81h-index23.0kcitations319works0.02yr avg
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Research Topics

Virus-based gene therapy research(113), Hemoglobinopathies and Related Disorders(91), CRISPR and Genetic Engineering(68), RNA Interference and Gene Delivery(47), Iron Metabolism and Disorders(29)

Publications316 total

Massive Myocardial Hemosiderosis
Case Reports in Cardiology·2023Open Access
High level of fetal-globin reactivation by designed transcriptional activator-like effector
Blood Advances·2020· 3 citedOpen Access
Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer
Blood·2018· 112 cited
George Stamatoyannopoulos (1934–2018)
Molecular Therapy·2018· 1 citedOpen Access
Gene Therapy for Hemophilia
Molecular Therapy·2017· 97 citedOpen Access
Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia
Molecular Therapy·2017· 6 citedOpen Access
Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector
Molecular Therapy — Methods & Clinical Development·2016· 13 citedOpen Access
686. Forced Fetal-Globin Reactivation Mediated by the TALE-ldb1 Fusion Protein
Molecular Therapy·2016Open Access
166. Lentiviral Hematopoietic Stem Cell Gene Therapy for Sjögren-Larsson Syndrome
Molecular Therapy·2016· 1 citedOpen Access
530. 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·2016· 2 citedOpen Access
Gene Therapy for Hemophilia
Human Gene Therapy·2016· 26 cited
<i>Blood</i>’s 70th anniversary: 1988-1992
Blood·2016Open Access
Hemoglobin Synthesis in Somatic Cell Hy Hybrids Between Mouse Erythroleukemia
2016
The identification of hematopoietic-specific regulatory elements for WASp gene expression
Molecular Therapy — Methods & Clinical Development·2016· 3 citedOpen Access
Mechanism of DNase I hypersensitive site formation within the human globin locus control region (hemoglobin switching/chromatin structure/erythroid specificity/transcription factor GATA-1/transcription factor NF-E2)
2016
289. Safe and Effective Gene Therapy for Wiskott – Aldrich Syndrome Using an Insulated Lentiviral Vector
Molecular Therapy·2015Open Access
555. Distribution of AAV8 Particles in Cell Lysates and Supernatants Changes with Time and Is Dependent on the Vector Genome
Molecular Therapy·2015Open Access
Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy
Molecular Therapy — Methods & Clinical Development·2015· 27 citedOpen Access
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
New England Journal of Medicine·2014· 1.2k citedOpen Access
Our Journey to Successful Gene Therapy for Hemophilia B
Human Gene Therapy·2014· 14 citedOpen Access
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Frequent Co-authors

Amit C. Nathwani(8), Derek A. Persons(7), Catherine Y. Ng(6), James A. Allay(6), John T. Gray(6), Andrew M. Davidoff(6), Hideki Hanawa(6), Elio F. Vanin(6), Brian P. Sorrentino(5), Cecilia Rosales(4), Jenny McIntosh(4), Junfang Zhou(4), Katherine A. High(4), Cynthia E. Dunbar(4), Edward G. D. Tuddenham(3), Christopher L. Morton(3), Susan Sleep(3), Federico Mingozzi(3), Deepak B. Thimiri Govinda Raj(3), Neal S. Young(3)