| First Author | Medina DX | Year | 2020 |
| Journal | Front Bioeng Biotechnol | Volume | 8 |
| Pages | 224 | PubMed ID | 32292776 |
| Mgi Jnum | J:357798 | Mgi Id | MGI:7763780 |
| Doi | 10.3389/fbioe.2020.00224 | Citation | Medina DX, et al. (2020) Intravenously Administered, Retinoid Activating Nanoparticles Increase Lifespan and Reduce Neurodegeneration in the SOD1(G93A) Mouse Model of ALS. Front Bioeng Biotechnol 8:224 |
| abstractText | Dysregulation of the retinoic acid (RA) signaling pathway is observed in amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. Here, we investigated the therapeutic potential of retinoid activation via the RA receptor beta (RARbeta) in the SOD1 (G93A) mouse model of ALS. Our approach utilized the RARbeta agonist adapalene, which we previously found to be neuroprotective in vitro. Adapalene, like most retinoids, is poorly water soluble, which has thus far prevented effective drug delivery in vivo. To address this challenge, we encapsulated adapalene within nanoparticles (Adap-NPs) composed of poly(lactic acid)-poly(ethylene glycol) (PLA-PEG). Our data demonstrate that intravenous administration of Adap-NPs robustly activates retinoid signaling in the CNS. Chronic administration of Adap-NPs resulted in improved motor performance, prolonged lifespan, and neuroprotection in SOD1 (G93A) mice. This study highlights retinoid signaling as a valuable therapeutic approach and presents a novel nanoparticle platform for the treatment of ALS. |
