Open AccessBiodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles
Author(s) -
Xavier Palazzi,
Ingrid Pardo,
Madhu P. Sirivelu,
Leah Newman,
Steven W. Kumpf,
Jessie Qian,
Tania Franks,
Sarah Lopes,
June Liu,
Laura Monarski,
Sandra Casinghino,
Casey Ritenour,
Hayley N Ritenour,
Christopher DuBois,
Olson Ja,
John Graves,
Kristin Alexander,
Timothy Coskran,
Thomas A. Lanz,
Joseph T. Brady,
Douglas M. McCarty,
Suryanarayan Somanathan,
Laurence O. Whiteley
Publication year - 2022
Publication title -
human gene therapy
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.633
H-Index - 149
eISSN - 1557-7422
pISSN - 1043-0342
DOI - 10.1089/hum.2021.116
Subject(s) - smn1 , transgene , biology , necrosis , hepatocyte , pathology , toxicity , pharmacology , medicine , gene , motor neuron , biochemistry , disease , in vitro