Results 31 to 40 of about 12,392,515 (266)
Adenovirus-based gene therapy approaches for hemophilia B [PDF]
, 2012 Gene therapy can be used to treat devastating inherited diseases, especially diseases and patients that are not suitable for a conventional cure. The blood clotting disorder hemophilia is one of the most extensively studied monogenetic diseases in gene ...
Zhang, Wenli
core +1 more source
Molecular Therapy: Methods & Clinical Development, 2017 Gene therapy during neonatal and infant stages is a promising approach for hemophilia B, a congenital disorder caused by deficiency of blood coagulation factor IX (FIX).
Shunsuke Iizuka +4 more
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The molecular basis of hemophilia B [PDF]
, 2022 Hemophilia B (HB) is a genetically determined bleeding disorder characterized by deficiency of the coagulation factor IX (FIX). The severity of bleeding phenotype is associated with FIX plasma level.
Odnoczko, Edyta; Department of Hemostasis and Metabolic Disorders, Institute of Hematology and Transfusion Medicine, Warsaw +2 more
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Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX
Cell Reports, 2018 Summary: Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient ...
Suvasini Ramaswamy +7 more
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Hemophilia-related health problems reported in the PROBE questionnaire (N = 25).
, 2023 Hemophilia-related health problems reported in the PROBE questionnaire (N = 25).
Raul Gamarra (16503189) +9 more
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Haemophilia B: an illustrative review of current challenges and opportunities
Research and Practice in Thrombosis and HaemostasisBackground: Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX, which presents unique challenges in clinical management.
Cedric Hermans +3 more
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Immune tolerance induction in a severe hemophilia B child with low titer inhibitors
Journal of Applied Hematology, 2022 Hemophilia B is an X-linked inherited bleeding disorder caused by either the absence or reduced biosynthesis of clotting factor IX (FIX). This disorder affects approximately 1 in 30,000 male individuals worldwide. Patients with severe form (FIX
Asia Almulla, Najam Awan, Faisl Khanani
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Chinese Expert Consensus on Pharmacokinetics Guided Treatment for Hemophilia A
, 2022 Hemophilia A is an X-chromosome-linked recessive genetic disease that lacks coagulation factor Ⅷ (Factor Ⅷ, FⅧ) and is clinically manifested as spontaneous or excessive bleeding after injury.The current main treatment for hemophilia A is alternative ...
Hemophilia Treatment Center Collaborative Network of China
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Cardiac surgery in patients with Hemophilia:is it safe?
Journal of Cardiothoracic Surgery, 2020 Background The life expectancy of hemophiliacs is similar to that of the general population. As a result, the prevalence of age-related cardiovascular diseases has increased.
Amjad Shalabi +9 more
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Engineering Biology Beyond Single Genes: Advances and Challenges in Multiplex Genome Editing
Animal Research and One Health, EarlyView.Multiplex genome editing is transforming genome engineering from single‐gene perturbation to network‐level control, yet its broader application remains limited by challenges in gRNA array engineering, delivery technologies, and safety management. Emerging AI‐driven approaches are accelerating guide RNA design and CRISPR effector optimization for ...
Linli Wang, Yongbin Liu, Hongbing Han
wiley +1 more source