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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...
Genome-wide Association Studies-GWAS01:11

Genome-wide Association Studies-GWAS

Genome-wide association studies or GWAS are used to identify whether common SNPs are associated with certain diseases. Suppose specific SNPs are more frequently observed in individuals with a particular disease than those without the disease. In that case, those SNPs are said to be associated with the disease. Chi-square analysis is performed to check the probability of the allele likely to be associated with the disease.
GWAS does not require the identification of the target gene involved in...
The JAK-STAT Signaling Pathway01:20

The JAK-STAT Signaling Pathway

Several cytokine receptors have tightly bound Janus kinase or JAK proteins attached at their cytosolic tail. Small signaling molecules such as cytokines, growth hormones, or prolactins bind to the cytokine receptors and initiate their dimerization. The dimerization brings the cytosolic JAKs together that trans-phosphorylate and activates each other. The activated JAKs now phosphorylate cytosolic tails of the cytokine receptors, which serve as binding sites for adaptor proteins such as  SH2...
T Cell Types and Functions01:24

T Cell Types and Functions

When T cells with CD4 markers are activated, they give rise to two types of effector cells: helper T cells and regulatory T cells. Meanwhile, T cells with CD8 markers differentiate into effector cytotoxic T cells. The differentiation of CD4 T cells into helper T cell subsets, such as Th1, Th2, and Th17 cells, is dependent on the antigen type, antigen-presenting cell, and regulatory cytokines.
Th1 cells stimulate dendritic cells to express necessary co-stimulatory molecules on their surfaces for...
Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF01:24

Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF

Tumor Necrosis Factor (TNF), a proinflammatory cytokine, contributes significantly to the inflammation seen in Crohn's disease. It exists as soluble TNF and membrane-bound TNF, with actions mediated through TNF receptors (TNFR). TNFR activation leads to the release of proinflammatory cytokines, T-cell activation, collagen production, and leukocyte migration, all contributing to inflammation in Crohn's disease. Anti-TNF monoclonal antibodies, namely infliximab (Remicade), adalimumab (Humira),...

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Related Experiment Video

Updated: Jul 19, 2026

Generation of Induced-pluripotent Stem Cells Using Fibroblast-like Synoviocytes Isolated from Joints of Rheumatoid Arthritis Patients
09:31

Generation of Induced-pluripotent Stem Cells Using Fibroblast-like Synoviocytes Isolated from Joints of Rheumatoid Arthritis Patients

Published on: October 16, 2016

Gene therapy for rheumatoid arthritis.

J N Gouze1, S C Ghivizzani, E Gouze

  • 1Department of Orthopedic Surgery, Harvard Medical School, Boston, MA, USA.

Hand Surgery : an International Journal Devoted to Hand and Upper Limb Surgery and Related Research : Journal of the Asia-Pacific Federation of Societies for Surgery of the Hand
|March 20, 2002
PubMed
Summary

Gene therapy shows promise for rheumatoid arthritis (RA) treatment. A Phase I trial successfully delivered anti-arthritic genes to human joints, demonstrating safety and paving the way for future orthopedic applications.

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Last Updated: Jul 19, 2026

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An Adoptive Transfer Model of Rheumatoid Arthritis in Mice
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An Adoptive Transfer Model of Rheumatoid Arthritis in Mice

Published on: June 6, 2025

Area of Science:

  • Orthopedics
  • Rheumatology
  • Gene Therapy

Background:

  • Rheumatoid arthritis (RA) is a chronic autoimmune disease causing joint inflammation and damage.
  • Advances in RA biology have revealed new therapeutic targets and strategies.
  • Gene therapy offers a novel approach by delivering therapeutic genes to affected tissues.

Purpose of the Study:

  • To evaluate the safety and feasibility of gene therapy in human joints for rheumatoid arthritis.
  • To assess the delivery of genes encoding anti-arthritic proteins in patients undergoing orthopedic surgery.

Main Methods:

  • A Phase I clinical trial was conducted involving subjects with RA.
  • A gene encoding interleukin-1 receptor antagonist (a potential anti-arthritic protein) was delivered to the metacarpophalangeal joints.
  • Gene transfer was performed one week prior to total joint arthroplasty.

Main Results:

  • The study successfully demonstrated the safe transfer of genes to human joints.
  • The gene therapy approach was well-tolerated in the trial participants.
  • This marks the first successful human Phase I trial of gene therapy for RA in joints.

Conclusions:

  • Gene therapy is a safe and feasible treatment option for rheumatoid arthritis in human joints.
  • This pioneering study supports further investigation of gene therapy for arthritis and other orthopedic conditions.
  • The findings open new therapeutic avenues for managing joint diseases.