نتائج البحث

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)

• Source: First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic

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A novel homozygous p.Ser69Pro SOD1 mutation causes severe young-onset ALS with decreased enzyme activity

• Authors : Fahmy, Nagia; Müller, Kathrin; Andersen, Peter Munch, 1962

Subjects: Enzyme activity; Homozygous; Novel mutation

• Source: Journal of Neurology. 270:1770-1773

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MS785-MS27 Reactive Misfolded/Non-Native Zn-Deficient SOD1 Species Exhibit Cytotoxicity and Adopt Heterozygous Conformations in Motor Neurons.

• Authors : Tokuda, Eiichi¹ (AUTHOR) ; Sakashita, Yume¹ (AUTHOR); Tokoro, Naoya¹ (AUTHOR)

Subjects: *MOTOR neurons; *CYTOTOXINS; *AMYOTROPHIC lateral sclerosis

• Source: International Journal of Molecular Sciences. Jun2024, Vol. 25 Issue 11, p5603. 21p.

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Nuclear Localization of Human SOD1 in Motor Neurons in Mouse Model and Patient Amyotrophic Lateral Sclerosis: Possible Links to Cholinergic Phenotype, NADPH Oxidase, Oxidative Stress, and DNA Damage.

• Authors : Martin, Lee J.^1,2,3,4 (AUTHOR) ; Koh, Shannon J.^1,5 (AUTHOR); Price, Antionette¹ (AUTHOR)

Subjects: *SINGLE-strand DNA breaks; *CENTRAL nervous system; *MOTOR neurons

• Source: International Journal of Molecular Sciences. Aug2024, Vol. 25 Issue 16, p9106. 25p.

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Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

• Source: A Phase 1/2, Multicenter, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With

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Adipose mesenchymal stem cells-derived extracellular vesicles exert their preferential action in damaged central sites of SOD1 mice rather than peripherally

• Authors : Ermanna Turano; Federica Virla; Ilaria Scambi

Subjects: Amyotrophic lateral sclerosis; extracellular vesicles; adipose-derived stem cells

• Source: European Journal of Histochemistry, Vol 68, Iss 3 (2024)

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Reduction of inflammation and mitochondrial degeneration in mutant SOD1 mice through inhibition of voltage-gated potassium channel Kv1.3.

• Authors : Ratano, Patrizia; Cocozza, Germana; Pinchera, Cecilia

Subjects: ALS; Kv1.3 channels; inflammation

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Respiratory onset of amyotrophic lateral sclerosis in a pregnant woman with a novel SOD1 mutation

• Authors : Masrori, Pegah; Ospitalieri, Simona; Forsberg, Karin

Subjects: antisense oligonucleotides; gene silencing; novel mutation

• Source: European Journal of Neurology. 29(4):1279-1283

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Amyloidogenic regions in beta-strands II and III modulate the aggregation and toxicity of SOD1 in living cells

• Authors : Luke McAlary; Jeremy R. Nan; Clay Shyu

Subjects: protein aggregation; protein homeostasis; amyotrophic lateral sclerosis

• Source: Open Biology, Vol 14, Iss 6 (2024)

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Protein aggregation and therapeutic strategies in SOD1- and TDP-43- linked ALS

• Authors : Maria Tsekrekou; Maria Giannakou; Katerina Papanikolopoulou

Subjects: amyotrophic lateral sclerosis; SOD1; TDP-43

• Source: Frontiers in Molecular Biosciences, Vol 11 (2024)

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