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Gene Therapy

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 Therapy

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...

Larynx

Larynx

The human larynx, often referred to as the voice box, is an intricate organ located in the neck. It serves as a pathway for air to enter the lungs during respiration and is an essential component of voice production.
Anatomy of the Larynx
The larynx consists of various components, including cartilage, muscles, and vocal cords. Its structure includes three large unpaired cartilages—the thyroid, cricoid, and epiglottis—and three smaller paired cartilages—the arytenoids, corniculates, and...

Myasthenia Gravis: Overview and Treatment

Myasthenia Gravis: Overview and Treatment

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Myasthenia Gravis ll: Pathophysiology

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The disease process of myasthenia gravis begins at the neuromuscular junction, where antibodies attack key proteins needed for muscle activation. This immune reaction weakens signal transmission, leading to the characteristic muscle fatigue and weakness that define the condition.Immune-Mediated DamageIn most individuals, antibodies target acetylcholine receptors (AChRs) on the postsynaptic membrane of muscle cells. By blocking acetylcholine binding, these antibodies prevent the nerve signal...

Skeletal Muscle Relaxants: Therapeutic Uses

Skeletal Muscle Relaxants: Therapeutic Uses

Skeletal muscle relaxants are used to relax muscle tone and alleviate painful muscle contractions. However, the choice of skeletal muscle relaxants depends on the duration of the surgical procedure in order to minimize potential side effects. Skeletal muscle relaxants like neuromuscular blocking agents [NMBAs] are commonly employed as adjuvants alongside general anesthetics in clinical settings. NMBAs are also used to maintain controlled ventilation during surgery of the larynx or pharynx as...

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Related Experiment Video

Updated: Jul 16, 2026

An Implantable System For Chronic In Vivo Electromyography

An Implantable System For Chronic In Vivo Electromyography

Published on: April 21, 2020

Gene therapy for laryngeal paralysis.

Akihiro Shiotani¹, Koichiro Saito, Koji Araki

¹Department of Otolaryngology-Head and Neck Surgery, National Defense Medical College, Saitama, Japan.

The Annals of Otology, Rhinology, and Laryngology

|March 29, 2007

Summary

Gene therapy shows promise for restoring dynamic vocal fold function in laryngeal paralysis. Glial cell line-derived neurotrophic factor (GDNF) gene transfer improved nerve recovery and vocal fold motion in rat models.

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Subcutaneous Neurotrophin 4 Infusion Using Osmotic Pumps or Direct Muscular Injection Enhances Aging Rat Laryngeal Muscles

Subcutaneous Neurotrophin 4 Infusion Using Osmotic Pumps or Direct Muscular Injection Enhances Aging Rat Laryngeal Muscles

Published on: June 13, 2017

Area of Science:

Regenerative Medicine
Gene Therapy
Neuroscience

Background:

Current surgical treatments for laryngeal paralysis offer limited functional recovery.
Laryngeal reinnervation procedures have not fully restored dynamic laryngeal function.
A novel approach is needed for neurologic restoration and regeneration in laryngeal paralysis.

Purpose of the Study:

To investigate the potential of gene therapy for laryngeal paralysis.
To assess the therapeutic effects of gene therapy in restoring dynamic laryngeal function.
To evaluate gene transfer of glial cell line-derived neurotrophic factor (GDNF) in preclinical models.

Main Methods:

Utilized rat models of laryngeal paralysis (vagal nerve avulsion and recurrent laryngeal nerve crush).
Adenovirus-mediated transfer of the GDNF gene into the nucleus ambiguus or recurrent laryngeal nerve fibers.
Investigated neuroprotective effects and functional recovery at 2 and 4 weeks post-gene transfer.

Main Results:

GDNF gene transfer significantly increased motoneuron survival in the vagal nerve avulsion model.
Simultaneous GDNF and brain-derived neurotrophic factor gene transfer enhanced neuroprotection.
GDNF gene transfer accelerated nerve conduction velocity and improved vocal fold motion in the crush model.

Conclusions:

Gene therapy, specifically using GDNF, presents a potential future treatment for laryngeal paralysis.
These findings suggest a pathway toward restoring dynamic laryngeal function.
Further research is required to confirm vector safety for clinical application.