☆ 4.3 Review

Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review

HAEMOPHILIA (2023)

Related references

Note: Only part of the references are listed.

Article Hematology

Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy

Annette von Drygalski et al.

Summary: This study reports the 3-year outcomes of using Etranacogene dezaparvovec in the treatment of adults with hemophilia B. The results show that Etranacogene dezaparvovec significantly increases FIX activity, reduces bleeding frequency, improves joint health, and has no major adverse events over the 3-year period.

BLOOD ADVANCES (2023)

Add to Collection

Article Medicine, General & Internal

Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B

Jinesh Shah et al.

Summary: This study predicts the long-term durability of factor IX activity levels in patients with hemophilia B receiving etranacogene dezaparvovec gene therapy. The results suggest that most patients would have sustained factor IX activity levels and would not require regular factor replacement products for up to 25.5 years following a single treatment.

CURRENT MEDICAL RESEARCH AND OPINION (2023)

Add to Collection

Article Medicine, General & Internal

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

S. W. Pipe et al.

Summary: Etranacogene dezaparvovec gene therapy was found to be superior to prophylactic factor IX treatment in patients with hemophilia B with regards to the annualized bleeding rate, and it demonstrated a favorable safety profile.

NEW ENGLAND JOURNAL OF MEDICINE (2023)

Add to Collection

Article Medicine, General & Internal

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

J. Mahlangu et al.

Summary: This study demonstrates the long-term effectiveness and safety of valoctocogene roxaparvovec therapy in reducing bleeding events in men with severe hemophilia A. The pharmacokinetic modeling suggests a half-life of 123 weeks for the transgene-derived factor VIII production system. Models also indicate a similar relationship between factor VIII activity and bleeding episodes as observed in individuals with mild-to-moderate hemophilia A using epidemiologic data.

NEW ENGLAND JOURNAL OF MEDICINE (2023)

Add to Collection

Letter Hematology

Gene therapy of hemophilia: Hub centres should be haemophilia centres: A joint publication of EAHAD and EHC

Wolfgang Miesbach et al.

HAEMOPHILIA (2022)

Add to Collection

Review Biotechnology & Applied Microbiology

Durability of transgene expression after rAAV gene therapy

Manish Muhuri et al.

Summary: Recombinant adeno-associated virus (rAAV) gene therapy has the potential to improve the lives of patients with genetic disorders. However, it is uncertain how long the therapeutic effect will last. This review explores the non-immunological and immunological factors that may affect durability, as well as strategies to increase the duration of the therapeutic effect.

MOLECULAR THERAPY (2022)

Add to Collection

Article Medicine, General & Internal

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

M. C. Ozelo et al.

Summary: The study evaluated the efficacy and safety of valoctocogene roxaparvovec in men with severe hemophilia A, showing an increase in factor VIII activity level, reduced bleeding rates, and factor VIII concentrate use.

NEW ENGLAND JOURNAL OF MEDICINE (2022)

Add to Collection

Review Biochemistry & Molecular Biology

The Arrival of Gene Therapy for Patients with Hemophilia A

Giancarlo Castaman et al.

Summary: The standard treatment for hemophilia A has been intravenous administration of exogenous factor VIII (FVIII), which has negative impact on patient's quality of life. The development of gene therapy brings hope for a single intravenous treatment that could result in long-term expression of FVIII and reduce bleeding episodes. Several gene therapies have shown positive outcomes in clinical trials and are expected to be approved for use in the near future.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2022)

Add to Collection

Review Chemistry, Medicinal

Manufacturing challenges and rational formulation development for AAV viral vectors

Arvind Srivastava et al.

Summary: AAV has emerged as a leading platform for gene delivery due to its safety profile and efficient transduction, but faces challenges in large-scale production and long-term storage. This review analyzes the challenges encountered during AAV vector manufacturing and discusses strategies to maintain product quality throughout shelf-life, highlighting the importance of understanding degradation mechanisms and formulation techniques.

JOURNAL OF PHARMACEUTICAL SCIENCES (2021)

Add to Collection

Review Hematology

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

Frank W. G. Leebeek et al.

Summary: Significant progress has been made in gene therapy for hemophilia A and B in the last decade, showing promising results in reducing bleeding episodes and prophylaxis needs for patients. However, challenges such as side effects and limitations still exist and need to be addressed before widespread application in the patient population.

BLOOD (2021)

Add to Collection

Article Hematology

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

K. John Pasi et al.

Summary: Valoctocogene roxaparvovec gene therapy has shown sustained clinical benefit in individuals with severe haemophilia A, with most participants maintaining FVIII levels and experiencing improved quality of life over a 5-year and 4-year period.

HAEMOPHILIA (2021)

Add to Collection

Article Hematology

Delivery of AAV-based gene therapy through haemophilia centres-A need for re-evaluation of infrastructure and comprehensive care: A Joint publication of EAHAD and EHC

Wolfgang Miesbach et al.

Summary: This article outlines the certification process offered to haemophilia centres in Europe by the European Haemophilia Network based on the number of patients and facilities. The aim is to reassess the infrastructure of haemophilia centres in order to implement criteria for gene therapy care in the future.

HAEMOPHILIA (2021)

Add to Collection

Review Hematology

Hemophilia Gene Therapy: Approaching the First Licensed Product

Paul Batty et al.

Summary: The clinical potential of hemophilia gene therapy has been pursued for the past 30 years, with expectations of achieving this goal in the near future. While recent late phase clinical trials have shown promising results, there are still issues regarding efficacy and safety that need further attention. Overcoming these challenges is crucial for the widespread application of this treatment paradigm.

HEMASPHERE (2021)

Add to Collection

Review Biotechnology & Applied Microbiology

Moving from the bench towards a large scale, industrial platform process for adeno-associated viral vector purification

Benjamin Adams et al.

BIOTECHNOLOGY AND BIOENGINEERING (2020)

Add to Collection

Article Hematology

WFH Guidelines for the Management of Hemophilia, 3rd edition

Alok Srivastava et al.

HAEMOPHILIA (2020)

Add to Collection

Article Medicine, General & Internal

Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males A Meta-analytic Approach Using National Registries

Alfonso Iorio et al.

ANNALS OF INTERNAL MEDICINE (2019)

Add to Collection

Review Hematology

The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world

Glenn F. Pierce et al.

HAEMOPHILIA (2019)

Add to Collection

Article Medicine, General & Internal

Emicizumab Prophylaxis in Hemophilia A with Inhibitors

Johannes Oldenburg et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Add to Collection

Review Oncology

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

Michael F. Naso et al.

BIODRUGS (2017)

Add to Collection

Article Hematology

Human liver sinusoidal endothelial cells but not hepatocytes contain factor VIII

T. Shahani et al.

JOURNAL OF THROMBOSIS AND HAEMOSTASIS (2014)

Add to Collection

Article Medicine, Research & Experimental

Hepatocyte Is a Sole Cell Type Responsible for the Production of Coagulation Factor IX In Vivo

Kohei Tatsumi et al.

CELL MEDICINE (2012)

Add to Collection

Review Genetics & Heredity

Production, Purification and Characterization of Adeno-Associated Vectors

Eduard Ayuso et al.

CURRENT GENE THERAPY (2010)

Add to Collection

Article Medicine, General & Internal

X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)

Paolo Simioni et al.

NEW ENGLAND JOURNAL OF MEDICINE (2009)

Add to Collection

Article Biochemistry & Molecular Biology

Current issues in adeno-associated viral vector production

OW Merten et al.

GENE THERAPY (2005)

Add to Collection

Review Biochemistry & Molecular Biology

Intracellular trafficking of adeno-associated viral vectors

W Ding et al.

GENE THERAPY (2005)

Add to Collection

© Peeref 2019-2024. All rights reserved.