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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Hemostasis is a crucial process that prevents excessive blood loss from damaged blood vessels. It involves various mechanisms such as vasoconstriction, platelet adhesion and activation, and fibrin formation. The importance of each mechanism depends on the type of vessel injury. In contrast, thrombosis is the abnormal formation of a blood clot within the blood vessels, leading to potential complications if the clot obstructs blood flow. Thrombosis can be caused by increased coagulability of the...
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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Pharmacogenomics: Identification of New Drug Targets01:29

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Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
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What is Genetic Engineering?00:49

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Disorders of Hemostasis01:24

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Hemostasis, the process that stops bleeding after a blood vessel injury, is crucial for maintaining the integrity of the circulatory system. However, disorders of hemostasis can disrupt this delicate balance, leading to either excessive clotting or bleeding. These disorders can be broadly classified into thromboembolic disorders and bleeding disorders.
Thromboembolic Disorders
Two factors primarily cause thromboembolic conditions.
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Related Experiment Video

Updated: Feb 23, 2026

Constitutive and Inducible Systems for Genetic In Vivo Modification of Mouse Hepatocytes Using Hydrodynamic Tail Vein Injection
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Gene Therapy for Hemophilia.

Amit C Nathwani1, Andrew M Davidoff2, Edward G D Tuddenham3

  • 1Department of Academic Haematology, UCL Cancer Institute, Katharine Dormandy Haemophilia and Thrombosis Centre, Rowland Hill Street, London NW3 2PF, United Kingdom; National Health Service Blood and Transplant, Oak House, Reeds Crescent, Watford, Hertfordshire, WD24 4QN, United Kingdom.

Hematology/Oncology Clinics of North America
|September 13, 2017
PubMed
Summary

Gene therapy offers a potential cure for hemophilia A and B by enabling continuous protein production, unlike current treatments requiring frequent, expensive infusions. This approach aims to provide a lasting solution for patients with factor VIII or factor IX deficiency.

Keywords:
Adeno-associated virus (AAV) vectorsClinical trialsGene therapyHemophilia

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Area of Science:

  • Hematology
  • Medical Genetics
  • Molecular Biology

Background:

  • Current hemophilia A and B treatments involve frequent intravenous infusions of costly clotting factors (factor VIII or factor IX).
  • These treatments result in suboptimal factor levels and breakthrough bleeding, with limited global access.
  • Existing therapies do not offer a cure, necessitating lifelong management.

Purpose of the Study:

  • To explore gene therapy as a curative approach for hemophilia A and B.
  • To investigate the potential for continuous endogenous expression of clotting factors.
  • To address the limitations of current protein replacement therapies.

Main Methods:

  • Gene therapy involves transferring a functional gene to replace the defective gene in hemophilic patients.
  • The goal is to induce sustained, endogenous production of factor VIII or factor IX.
  • This approach contrasts with the exogenous protein administration of current treatments.

Main Results:

  • Gene therapy holds the promise of continuous, endogenous expression of deficient clotting factors.
  • This could lead to sustained therapeutic levels, potentially eliminating the need for frequent infusions.
  • Successful gene therapy could represent a functional cure for hemophilia.

Conclusions:

  • Gene therapy presents a transformative alternative to current hemophilia treatments.
  • It offers the potential for a one-time curative intervention by restoring natural clotting factor production.
  • This approach could significantly improve the quality of life and long-term outcomes for hemophilia patients worldwide.