Autologous treatment for ALS with implication for broad neuroprotection.

ABSTRACT:

Background

Amyotrophic lateral sclerosis (ALS) is characterized by a progressive loss of motor neurons (MNs), leading to paralysis, respiratory failure and death within 2-5 years of diagnosis. The exact mechanisms of sporadic ALS, which comprises 90% of all cases, remain unknown. In familial ALS, mutations in superoxide dismutase (SOD1) cause 10% of cases.

Methods

ALS patient-derived human-induced pluripotent stem cells (ALS hiPSCs, harboring the SOD1^AV4 mutation), were differentiated to MNs (ALS-MNs). The neuroprotective effects of conditioned medium (CM) of hESCs (H9), wt hiPSCs (WTC-11) and the ALS iPSCs, on MN apoptosis and viability, formation and maintenance of neurites, mitochondrial activity and expression of inflammatory genes, were examined. For in vivo

SUBMITTER: Kim D 

PROVIDER: S-EPMC8915496 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature