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Microbiology

D-Index
51
Citations
12415
World Ranking
4290
National Ranking
383

Overview

Amit C. Nathwani is affiliated with University College London in the United Kingdom and specializes in research predominantly within the fields of biochemistry, genetics, and molecular biology, as well as medicine. Their scholarly output includes 34 publications in biochemistry, genetics, and molecular biology and 21 in medicine.

Their subfields of study emphasize molecular biology with 16 publications, genetics with 13, oncology with 9, hematology with 7, and physiology with 4. Their research covers main topics including virus-based gene therapy, CAR-T cell therapy, CRISPR and genetic engineering, hemophilia treatment, viral infectious diseases and gene expression in insects, lysosomal storage disorders, and transgenic plants and applications.

Frequent publication venues include Blood with 6 papers, Molecular Genetics and Metabolism with 3, Human Gene Therapy with 2, as well as one paper each in the New England Journal of Medicine and Hematology.

Key recent papers contributed by Amit C. Nathwani include:

  • Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B, 2022, New England Journal of Medicine
  • Gene therapy for hemophilia, 2022, Hematology
  • The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective, 2021, Blood
  • Liver Gene Therapy, 2022, Human Gene Therapy

Frequent co-authors working with Nathwani include Andrew M. Davidoff with 6 joint publications, Pratima Chowdary, Ulrike M. Reiss, Edward G. D. Tuddenham, and Rose Sheridan each with 5 joint publications.

Best Publications

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

    Amit C. Nathwani;Edward G.D. Tuddenham;Savita Rangarajan;Cecilia Rosales

  • 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

  • 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

  • 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

  • Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway.

    Andrew M. Davidoff;Catherine Y. C. Ng;Junfang Zhou;Yunyu Spence

  • Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models.

    Andrew M. Davidoff;John T. Gray;Catherine Y.C. Ng;Youbin Zhang

  • Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

    Jenny McIntosh;Jenny McIntosh;Peter J. Lenting;Cecilia Rosales;Cecilia Rosales;Doyoung Lee

  • Comparison of Various Envelope Proteins for Their Ability to Pseudotype Lentiviral Vectors and Transduce Primitive Hematopoietic Cells from Human Blood

    Hideki Hanawa;Patrick F Kelly;Amit C Nathwani;Derek A Persons

  • 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

  • Programmed death 1 expression during antiviral treatment of chronic hepatitis B: Impact of hepatitis B e‐antigen seroconversion

    Alexander Evans;Antonio Riva;Helen Cooksley;Sandra Phillips

  • Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

    Amit C. Nathwani;Andrew M. Davidoff;Hideki Hanawa;Yunyu Hu

  • Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA

    Amit C. Nathwani;Andrew Davidoff;Hideki Hanawa;Jun-Fang Zhou

  • Codon optimization of human factor VIII cDNAs leads to high-level expression

    Natalie J. Ward;Suzanne M. K. Buckley;Simon N. Waddington;Thierry VandenDriessche;Thierry VandenDriessche

  • A high-throughput sequencing test for diagnosing inherited bleeding, thrombotic, and platelet disorders

    Ilenia Simeoni;Ilenia Simeoni;Jonathan C. Stephens;Jonathan C. Stephens;Fengyuan Hu;Fengyuan Hu;Sri V V Deevi;Sri V V Deevi

  • Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein.

    Patrick F. Kelly;Jody Vandergriff;Amit Nathwani;Arthur W. Nienhuis

  • Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial.

    James A. Allay;Susan Sleep;Scott Long;David M. Tillman

  • Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock

    Andrew M. Davidoff;Catherine Y.C. Ng;Susan Sleep;John Gray

  • Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

    Nicole K. Paulk;Katja Pekrun;Erhua Zhu;Sean Nygaard

  • Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8.

    M Natkunarajah;P Trittibach;J McIntosh;Y Duran

  • Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

    Mei Hong Tan;Alexander J. Smith;Basil Pawlyk;Xiaoyun Xu

Frequent Co-Authors

Arthur W. Nienhuis
Arthur W. Nienhuis St. Jude Children's Research Hospital
Simon N. Waddington
Simon N. Waddington University College London
Edward G. D. Tuddenham
Edward G. D. Tuddenham University College London
Adrian J. Thrasher
Adrian J. Thrasher University College London
Katherine A. High
Katherine A. High Children's Hospital of Philadelphia
Federico Mingozzi
Federico Mingozzi Children's Hospital of Philadelphia
Edward A. Clark
Edward A. Clark University of Washington
Peter M. Lydyard
Peter M. Lydyard University College London
Mark A. Kay
Mark A. Kay Stanford University
John H. McVey
John H. McVey University of Surrey

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