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Updated: Jan 7, 2026

Real-Time Fluorescent Measurement of Synaptic Functions in Models of Amyotrophic Lateral Sclerosis

Real-Time Fluorescent Measurement of Synaptic Functions in Models of Amyotrophic Lateral Sclerosis

Published on: July 16, 2021

Engineered GM1 Intersects Between Mitochondrial and Synaptic Pathways to Ameliorate ALS Pathology.

Federica Pilotto^1,2, Tristan Dellazizzo Toth^3,4, Silvano Bond^3,4

¹Institut Neuromyogène, Pathophysiology and genetics of the neuron and muscle, Inserm U1315, CNRS, Université Claude Bernard Lyon I, Lyon, France.

Advanced Science (Weinheim, Baden-Wurttemberg, Germany)

|January 5, 2026

Summary

Amyotrophic Lateral Sclerosis (ALS) involves motor neuron degeneration. A new nanoliposome formulation of GM1 ganglioside, Talineuren (TLN), shows promise by restoring cellular functions and counteracting deficits in ALS mouse models.

Keywords:

C9ORF72 ER‐mitochondria contacts GM1 SOD1‐G93A amyotrophic lateral sclerosis (ALS)mitochondrial dysfunction nanoliposomal formulation proteomics synaptic dysfunction

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Area of Science:

Neuroscience
Genetics
Biochemistry

Background:

Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by motor neuron loss.
Genetic heterogeneity in ALS presents challenges for developing effective treatments.
Identifying conserved molecular pathways is crucial for advancing ALS therapeutics.

Purpose of the Study:

To investigate conserved pathogenic mechanisms in C9ORF72-linked ALS using a cross-species proteomic approach.
To evaluate the neuroprotective potential of GM1 ganglioside and its nanoliposome formulation, Talineuren (TLN), in ALS models.

Main Methods:

Proteomic analysis of human iPSC-derived motor neurons and mouse spinal cords with C9ORF72 mutations.
Assessment of synaptic vesicle release, endoplasmic reticulum (ER) and mitochondrial stress responses.
Pharmacokinetic evaluation and therapeutic efficacy testing of Talineuren (TLN) in C9ORF72 and SOD1-G93A mouse models.

Main Results:

Conserved disruptions in synaptic vesicle release, ER, and mitochondrial stress were identified as key ALS mechanisms.
Disease progression correlated with protein aggregate accumulation and oxidative stress.
Talineuren (TLN) improved mitochondrial function, alleviated ER stress, prevented protein aggregation, and restored proteostasis.
Talineuren (TLN) counteracted behavioral deficits in ALS mouse models.

Conclusions:

Cellular disruptions, including ER and mitochondrial dysfunction, play a central role in ALS pathogenesis.
Talineuren (TLN), a bioavailable formulation of GM1 ganglioside, demonstrates significant neuroprotective effects.
Talineuren (TLN) represents a promising therapeutic candidate for ALS treatment.