Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

por Pourshafie, Naemeh, Lee, Philip R., Chen, Ke-lian, Harmison, George G., Bott, Laura C., Fischbeck, Kenneth H., Rinaldi, Carlo
Publicado 2018

MicroRNA-298 reduces levels of human amyloid-β precursor protein (APP), β-site APP-converting enzyme 1 (BACE1) and specific tau protein moieties

por Chopra, Nipun, Wang, Ruizhi, Maloney, Bryan, Nho, Kwangsik, Beck, John S., Pourshafie, Naemeh, Niculescu, Alexander, Saykin, Andrew J., Rinaldi, Carlo, Counts, Scott E., Lahiri, Debomoy K.
Publicado 2020

MiR-298 Counteracts Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular Atrophy

por Pourshafie, Naemeh, Lee, Philip R, Chen, Ke-lian, Harmison, George G, Bott, Laura C, Katsuno, Masahisa, Sobue, Gen, Burnett, Barrington G, Fischbeck, Kenneth H, Rinaldi, Carlo
Publicado 2016

LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice

por Prakasam, Ramachandran, Bonadiman, Angela, Andreotti, Roberta, Zuccaro, Emanuela, Dalfovo, Davide, Marchioretti, Caterina, Tripathy, Debasmita, Petris, Gianluca, Anderson, Eric N., Migazzi, Alice, Tosatto, Laura, Cereseto, Anna, Battaglioli, Elena, Sorarù, Gianni, Lim, Wooi Fang, Rinaldi, Carlo, Sambataro, Fabio, Pourshafie, Naemeh, Grunseich, Christopher, Romanel, Alessandro, Pandey, Udai Bhan, Contestabile, Andrea, Ronzitti, Giuseppe, Basso, Manuela, Pennuto, Maria
Publicado 2023

Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity

por Lim, Wooi F., Forouhan, Mitra, Roberts, Thomas C., Dabney, Jesse, Ellerington, Ruth, Speciale, Alfina A., Manzano, Raquel, Lieto, Maria, Sangha, Gavinda, Banerjee, Subhashis, Conceição, Mariana, Cravo, Lara, Biscans, Annabelle, Roux, Loïc, Pourshafie, Naemeh, Grunseich, Christopher, Duguez, Stephanie, Khvorova, Anastasia, Pennuto, Maria, Cortes, Constanza J., La Spada, Albert R., Fischbeck, Kenneth H., Wood, Matthew J.A., Rinaldi, Carlo
Publicado 2021