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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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Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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CRISPR01:59

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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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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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Symptoms, Diagnosis, and Treatment for Women and Girls With Hemophilia: A Narrative Review.

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External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities.

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Hemophilia gene therapy: first, do no harm.

Leonard A Valentino1, Radoslaw Kaczmarek2, Glenn F Pierce3

  • 1Rush University, Chicago, Illinois, USA; National Hemophilia Foundation, New York, New York, USA.

Journal of Thrombosis and Haemostasis : JTH
|June 23, 2023
PubMed
Summary

Gene therapy offers new hope for hemophilia treatment, but requires careful consideration due to historical safety concerns. Informed decision-making is crucial for patients considering adeno-associated virus gene therapies for hemophilia A and B.

Keywords:
efficacygene therapyharmhemophiliasafety

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Area of Science:

  • Hematology
  • Gene Therapy
  • Viral Vectors

Background:

  • The hemophilia community has a history of devastating infections (HIV, Hepatitis C) linked to past treatments like factor concentrates.
  • Despite advances, hemophilia treatment still faces challenges in survival, quality of life, and health equity.
  • Adeno-associated virus (AAV)-mediated, liver-directed gene therapy represents a significant advancement with both promise and uncertainty.

Purpose of the Study:

  • To review data supporting the regulatory authorization of AAV gene therapies for hemophilia.
  • To provide an overview of the gene therapy decision-making process for patients and healthcare professionals.
  • To recommend steps for ensuring safety and optimal outcomes in hemophilia gene therapy.

Main Methods:

  • Review of clinical trial data leading to regulatory approval of valoctocogene roxaparvovec (hemophilia A) and etranacogene dezaparvovec-drlb (hemophilia B).
  • Analysis of historical safety data related to hemophilia treatments.
  • Examination of shared decision-making principles in the context of novel gene therapies.

Main Results:

  • Valoctocogene roxaparvovec (AAV5 gene therapy) authorized in Europe for hemophilia A.
  • Etranacogene dezaparvovec-drlb authorized in US and Europe for hemophilia B.
  • Gene therapy offers potential for improved outcomes but necessitates a thorough, unbiased decision-making process.

Conclusions:

  • AAV-mediated gene therapy represents a promising new era in hemophilia treatment.
  • Informed, shared decision-making is paramount for patients considering gene therapy.
  • Addressing potential biases and implementing safety protocols are essential for successful gene therapy adoption in hemophilia care.