Researchers found that injecting a gene producing insulin-like growth factor-1 (IGF-1) into muscles delayed disease onset by 31 days, preserved nerve cells, and reduced muscle wasting. This study may lead to a new, gene-based treatment for ALS affecting over 30,000 Americans.
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Researchers at Johns Hopkins and Salk Institute develop gene therapy that slows progression of amyotrophic lateral sclerosis (ALS), a devastating disease affecting thousands. In experiments with mice, injection of insulin-like growth factor-1 (IGF-1) into muscles extends survival and improves strength.
Researchers at Project A.L.S. have successfully tested a gene therapy approach that delays disease progression and prolongs survival in a mouse model of ALS. The treatment uses an adeno-associated viral vector to express the IGF1 protein, promoting neuronal survival.
A new three-year grant will help researchers better understand the causes and risk factors of ALS, a neurodegenerative disease that affects 30,000 people in the US. The study aims to investigate the link between a genetic mutation called Hfe and the onset and progression of ALS.
Researchers used 3D imaging to study ALS mutant proteins, finding they interact incorrectly and form toxic complexes that interfere with nerve cell function. The study supports two theories: oxidative damage from mutant SOD1 protein and aggregation of protein complexes.
Researchers have discovered a genetic link between specific mutations in the Dnchc1 gene and selective death of motor neurons, causing diseases like ALS. The study provides new insight into the pathogenetic mechanism underlying MND.
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Researchers identified chromosomal abnormalities in 5.9% of ALS patients, suggesting a previously unknown risk factor for the disease. The study highlights the need for systematic genetic analysis of patients with ALS and other neurodegenerative diseases.
Researchers discovered a gene, Hsp27, that protects mature nerve cells from death following injury. Delivering this gene via therapy may lead to new treatments for neurodegenerative diseases and nerve injuries.
Researchers have found that growth factors play a key role in regulating how embryonic nerve cells acquire the ability to process information. This study suggests possible avenues for treating damaged nerve cells and restoring their function.
Scientists have found a gene responsible for a rare form of hereditary spastic paraplegia, a condition that slowly robs children of their ability to walk. The discovery has opened the door to better diagnosis and treatment of the disorder, as well as insights into other spinal cord problems.
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A second gene mutation for the inherited form of ALS has been discovered by Northwestern University researcher Teepu Siddique. The newly identified gene, alsin, is responsible for juvenile inherited ALS (ALS2), a rare and slowly progressive disease affecting young populations in North Africa and the Middle East.