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Medical illustration of a scientist replacing part of a DNA molecule with tweezers, representing genome editing.
On the Hunt for the Next Breakthrough in Motor Neuron Disease
Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.
On the Hunt for the Next Breakthrough in Motor Neuron Disease
On the Hunt for the Next Breakthrough in Motor Neuron Disease

Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.

Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.

motor neuron disease

David Liu
Targeting a Genetic Accident to Treat Disease
Deanna MacNeil, PhD | May 9, 2023 | 2 min read
David Liu shares how integrating chemistry and evolution in his research has directed his work on base editing techniques aimed at developing new therapeutics.
A false color transmission electron microscope micrograph showing the nuclear envelope, the rough endoplasmic reticulum, the nucleus, and the cytoplasm.
New Gene Mutants Identified in Rare Motor Neuron Diseases
Clare Watson | Oct 17, 2022 | 2 min read
The discovery of gene variants in cases of hereditary spastic dysplasia could provide a diagnosis to affected families where no genetic cause could be found before.
Motor neurons, undergoing degeneration in ALS
Mutant T Cells That Drive Amyotrophic Lateral Sclerosis (ALS) Progression May React To a Brain Antigen
Nele Haelterman, PhD | Aug 8, 2022 | 4 min read
Scientists discovered a possibly autoreactive T cell population that forecasts and supports disease progression.
Electrode array, with needle-like electrodes facing upward
Brain Implant Allows Completely Paralyzed Patient to Communicate
Natalia Mesa, PhD | Mar 24, 2022 | 4 min read
The patient, who has ALS, is able to communicate in complete sentences by deliberately altering his brain’s activity.
neurological disorder, brain disease, Q&A, Alzheimer's disease, Parkinson's disease, multiple sclerosis, motor neuron disease, neurodegeneration, blood-brain barrier, sex
Sex of Human Cells Matters in Studying Neurological Disease
Amanda Heidt | Mar 18, 2021 | 5 min read
Authors of a new review urge researchers to consider how the chromosomal sex of a cell affects experiments in research on neurodegeneration.
Additional #IceBucketChallenge Payoffs
Jef Akst | Jul 28, 2016 | 2 min read
Researchers identify a new ALS-associated gene thanks to funds generated by the social media challenge that went viral in summer 2014.
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