Amit C. Nathwani

98 Chenies Mews, WC1E 6HX
London, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 81 publications

AAV Gene Therapy in Severe Hemophilia B. Reply.

AAV Gene Therapy in Severe Hemophilia B. Reply.

Journal: The New England journal of medicine
Published: August 20, 2025

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.

Journal: The New England journal of medicine
Published: June 11, 2025

A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy.

A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy.

Journal: Journal of thrombosis and haemostasis : JTH
Published: May 09, 2025

FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1.

FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: December 10, 2024

Alternative AAV gene therapy for hemophilia A using expression of Bi8, a novel single-chain FVIII-mimetic antibody.

Alternative AAV gene therapy for hemophilia A using expression of Bi8, a novel single-chain FVIII-mimetic antibody.

Journal: Blood
Published: November 15, 2024

Expression and treatment of ROR1+ cells with bispecific T-cell engagers in pediatric acute lymphoblastic leukemia.

Expression and treatment of ROR1+ cells with bispecific T-cell engagers in pediatric acute lymphoblastic leukemia.

Journal: Blood advances
Published: May 29, 2024

10 years of BiTE immunotherapy: an overview with a focus on pancreatic cancer.

10 years of BiTE immunotherapy: an overview with a focus on pancreatic cancer.

Journal: Frontiers in oncology
Published: May 07, 2024

Pre-Existing Immunity to a Nucleic Acid Contaminant-Derived Antigen Mediates Transaminitis and Resultant Diminished Transgene Expression in a Mouse Model of Hepatic Recombinant Adeno-Associated Virus-Mediated Gene Transfer.

Pre-Existing Immunity to a Nucleic Acid Contaminant-Derived Antigen Mediates Transaminitis and Resultant Diminished Transgene Expression in a Mouse Model of Hepatic Recombinant Adeno-Associated Virus-Mediated Gene Transfer.

Journal: Human gene therapy
Published: February 29, 2024

MD-1 downregulation is associated with reduced cell surface CD180 expression in CLL.

MD-1 downregulation is associated with reduced cell surface CD180 expression in CLL.

Journal: Leukemia research
Published: February 13, 2024

Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome.

Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome.

Journal: Science translational medicine
Published: August 09, 2023

Nebulized Recombinant Tissue Plasminogen Activator (rt-PA) for Acute COVID-19-Induced Respiratory Failure: An Exploratory Proof-of-Concept Trial.

Nebulized Recombinant Tissue Plasminogen Activator (rt-PA) for Acute COVID-19-Induced Respiratory Failure: An Exploratory Proof-of-Concept Trial.

Journal: Journal of clinical medicine
Published: June 19, 2023

Transplacental delivery of therapeutic proteins by engineered immunoglobulin G: a step toward perinatal replacement therapy.

Transplacental delivery of therapeutic proteins by engineered immunoglobulin G: a step toward perinatal replacement therapy.

Journal: Journal of thrombosis and haemostasis : JTH
Published: January 01, 2023
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Last Updated: 09/26/2026