Comprehensive evaluation of human-derived anti-poly-GA antibodies in cellular and animal models of C9orf72 disease.
Jambeau M, Meyer KD, Hruska-Plochan M, Tabet R, Lee CZ, Ray-Soni A, Aguilar C, Savage K, Mishra N, Cavegn N, Borter P, Lin CC, Jansen-West KR, Jiang J, Freyermuth F, Li N, De Rossi P, Pérez-Berlanga M, Jiang X, Daughrity LM, Pereira J, Narayanan S, Gu Y, Dhokai S, Dalkilic-Liddle I, Maniecka Z, Weber J, Workman M, McAlonis-Downes M, Berezovski E, Zhang YJ, Berry J, Wainger BJ, Kankel MW, Rushe M, Hock C, Nitsch RM, Cleveland DW, Petrucelli L, Gendron TF, Montrasio F, Grimm J, Polymenidou M, Lagier-Tourenne C.
Jambeau M, et al. Among authors: hock c.
Proc Natl Acad Sci U S A. 2022 Dec 6;119(49):e2123487119. doi: 10.1073/pnas.2123487119. Epub 2022 Dec 1.
Proc Natl Acad Sci U S A. 2022.
PMID: 36454749
Free PMC article.
Targeting poly-GA and poly-GP in two mouse models expressing G(4)C(2) repeats by systemic antibody delivery for up to 16 mo was well-tolerated and led to measurable brain penetration of antibodies. Long-term treatment with anti-GA antibodies produced improvement in an open …
Targeting poly-GA and poly-GP in two mouse models expressing G(4)C(2) repeats by systemic antibody delivery for up to 16 mo was well-tolerat …