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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...
iPS Cell Differentiation01:22

iPS Cell Differentiation

The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
Stem Cell Therapy for Tissue Regeneration01:21

Stem Cell Therapy for Tissue Regeneration

Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
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What is Genetic Engineering?

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Orthopedic gene therapy in 2008.

Christopher H Evans1, Steven C Ghivizzani, Paul D Robbins

  • 1Center for Molecular Orthopaedics, Harvard Medical School, Boston, Massachusetts, USA. cevans@bidmc.harvard.edu

Molecular Therapy : the Journal of the American Society of Gene Therapy
|December 11, 2008
PubMed
Summary

Gene therapy shows promise for treating orthopedic disorders like arthritis and bone injuries, offering effective solutions for debilitating conditions. Despite challenges, advancements suggest gene therapy could revolutionize orthopedic care, addressing unmet needs in patient treatment.

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Area of Science:

  • Orthopedics
  • Gene Therapy
  • Regenerative Medicine

Background:

  • Orthopedic disorders are a major cause of morbidity and socioeconomic burden, with increasing prevalence due to aging and obesity.
  • Many orthopedic conditions are challenging to treat with conventional methods, presenting an opportunity for novel therapeutic approaches.
  • Existing treatments for some orthopedic conditions are limited, necessitating the exploration of advanced interventions.

Purpose of the Study:

  • To explore the potential of gene therapy as a treatment modality for a range of orthopedic disorders.
  • To review the current status of gene therapy applications in orthopedics, including clinical trials and preclinical developments.
  • To identify barriers and future prospects for the clinical implementation of gene therapy in orthopedic care.

Main Methods:

  • Review of existing clinical trial data for gene therapy in arthritis and aseptic loosening of prosthetic joints.
  • Assessment of preclinical data for bone-healing applications and other orthopedic regenerative strategies.
  • Analysis of potential gene therapy targets including Mendelian diseases, orthopedic tumors, and tissue repair (cartilage, ligaments, tendons).

Main Results:

  • Clinical trials for arthritis and prosthetic joint loosening are underway, with bone-healing applications in advanced preclinical stages.
  • Gene therapy holds potential for treating genetic orthopedic diseases, tumors, and facilitating the regeneration of musculoskeletal tissues.
  • Preclinical and early clinical data demonstrate significant promise for gene therapy's efficacy in managing difficult orthopedic conditions.

Conclusions:

  • Gene therapy is a viable and promising approach for addressing several common and debilitating orthopedic disorders.
  • Despite regulatory and funding hurdles, existing technologies support the achievable clinical application of gene therapy in orthopedics.
  • Gene therapy offers a potentially straightforward and effective solution for orthopedic conditions that are currently difficult and expensive to treat.