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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 inserted. The...

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Gene delivery to bone.

¹Center for Advanced Orthopaedic Studies, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA 02215, USA. cevans@bidmc.harvard.edu

Advanced Drug Delivery Reviews

|April 7, 2012

Summary

This summary is machine-generated.

Gene delivery to bone offers a promising therapeutic strategy, enabling sustained protein expression for skeletal diseases. Research shows successful preclinical gene transfer to bone using various vectors and methods.

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Area of Science:

Biomedical Engineering
Molecular Biology
Regenerative Medicine

Background:

Gene delivery to bone serves as a valuable research tool and potential therapeutic approach.
Advantages over protein delivery include directed, sustained, and regulated expression of native proteins.

Purpose of the Study:

To review the preclinical literature on gene delivery to bone.
To highlight the potential of gene therapy for skeletal diseases and bone regeneration.

Main Methods:

Exploration of recombinant viral vectors (adenovirus, retrovirus, lentivirus) and non-viral vectors.
Investigation of ex vivo and in vivo gene delivery strategies, including gene-activated matrices.
Utilization of mesenchymal stem cells (MSCs) for ex vivo delivery and potential systemic bone targeting.

Main Results:

Substantial preclinical evidence demonstrates successful gene transfer and intraosseous expression.
Ex vivo delivery using MSCs shows potential for osteoblast differentiation and systemic skeletal disease treatment.
Local delivery of osteogenic transgenes, like bone morphogenetic proteins, shows promise for bone regeneration.

Conclusions:

Gene delivery to bone is a viable preclinical strategy with significant therapeutic potential.
Both ex vivo and in vivo approaches, particularly with MSCs and gene-activated matrices, are effective.
Targeted gene therapy holds promise for treating conditions like osteoporosis, osteogenesis imperfecta, bone defects, and avascular necrosis.