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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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.
What is Genetic Engineering?00:49

What is Genetic Engineering?

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Forced Transdifferentiation01:28

Forced Transdifferentiation

Transdifferentiation, also known as lineage reprogramming, was first discovered by Selman and Kafatos in 1974 in silkmoths. They observed that the moths’ cuticle-producing cells transformed into salt-producing cells. Many such cases of natural transdifferentiation occur in organisms. In humans, pancreatic alpha cells can become beta cells. In newts, the loss of the eye’s lens causes the pigmented epithelial cells to transdifferentiate into the lens cells.
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Related Experiment Video

Updated: May 23, 2026

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
11:16

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

Gene therapy, an ongoing revolution.

Olivier Benveniste1

  • 1Université Pierre et Marie Curie.

Blood
|March 31, 2012
PubMed
Summary
This summary is machine-generated.

Gene therapy using adeno-associated virus serotype 2 (AAV-2) showed long-term factor IX (FIX) expression for up to 10 years in a hemophilia B patient. This demonstrates sustained FIX production after AAV-2 gene delivery to muscles.

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Area of Science:

  • Hematology
  • Gene Therapy
  • Viral Vectors

Background:

  • Hemophilia B is a genetic bleeding disorder caused by deficiency of factor IX (FIX).
  • Current treatments involve regular infusions of FIX concentrate, posing challenges for patients.
  • Gene therapy offers a potential alternative for long-term FIX expression.

Purpose of the Study:

  • To evaluate the long-term persistence of factor IX (FIX) expression following adeno-associated virus serotype 2 (AAV-2) gene therapy in a patient with hemophilia B.
  • To assess the durability of FIX expression in muscles injected with AAV-2 vectors.

Main Methods:

  • A single patient with hemophilia B received intramuscular injections of AAV-2 carrying the FIX gene.
  • Factor IX levels and expression were monitored over a 10-year period post-injection.

Main Results:

  • Sustained expression of factor IX (FIX) was observed for up to 10 years after AAV-2 gene therapy.
  • The adeno-associated virus serotype 2 vector demonstrated long-term persistence of gene expression in injected muscles.

Conclusions:

  • Intramuscular AAV-2 gene therapy can lead to durable factor IX expression in hemophilia B patients.
  • This study highlights the potential of AAV-2 gene therapy for sustained treatment of hemophilia B.