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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...
Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
Alzheimer's Disease: Treatment01:22

Alzheimer's Disease: Treatment

Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...

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Related Experiment Video

Updated: May 29, 2026

Intramuscular Injections Along the Motor End Plates: A Minimally Invasive Approach to Shuttle Tracers Directly into Motor Neurons
10:57

Intramuscular Injections Along the Motor End Plates: A Minimally Invasive Approach to Shuttle Tracers Directly into Motor Neurons

Published on: July 13, 2015

Gene therapy for amyotrophic lateral sclerosis.

Thais Federici1, Nicholas M Boulis

  • 1Emory University, Department of Neurosurgery, 1365B Clifton Road NE, Suite B6200, Atlanta, GA 30322, USA.

Neurobiology of Disease
|September 6, 2011
PubMed
Summary

Gene therapy offers a promising avenue for amyotrophic lateral sclerosis (ALS). This review covers therapeutic transgenes, rodent models, and challenges for motor neuron disease treatments.

Area of Science:

  • Neuroscience
  • Genetics
  • Biomedical Engineering

Background:

  • Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease.
  • Current treatments for ALS are limited, necessitating novel therapeutic approaches.
  • Gene therapy presents a potential strategy for ALS treatment.

Purpose of the Study:

  • To review promising therapeutic transgenes for ALS.
  • To discuss proof-of-principle studies in transgenic rodent models of ALS.
  • To explore challenges and future directions in gene therapy for motor neuron diseases.

Main Methods:

  • Review of existing literature on gene therapy for ALS.
  • Analysis of studies utilizing transgenic rodent models.
  • Discussion of therapeutic targets and intervention timing.

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ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

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Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice
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Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice

Published on: February 24, 2026

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Last Updated: May 29, 2026

Intramuscular Injections Along the Motor End Plates: A Minimally Invasive Approach to Shuttle Tracers Directly into Motor Neurons
10:57

Intramuscular Injections Along the Motor End Plates: A Minimally Invasive Approach to Shuttle Tracers Directly into Motor Neurons

Published on: July 13, 2015

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
15:48

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

Published on: July 29, 2007

Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice
04:41

Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice

Published on: February 24, 2026

Main Results:

  • Identification of several promising therapeutic transgenes for ALS.
  • Demonstration of proof-of-principle for gene therapy in preclinical ALS models.
  • Highlighting of key challenges in translating gene therapy to clinical practice.

Conclusions:

  • Gene therapy holds significant potential for treating ALS.
  • Further research is needed to overcome challenges related to disease targets and intervention timing.
  • Gene therapy may also be applicable to other motor neuron diseases.