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Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles
Author(s) -
Xavier Palazzi,
Ingrid D. 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,
Jennifer Olson,
John Graves,
Kristin Alexander,
Timothy Coskran,
Thomas A. Lanz,
Joseph T. Brady,
Douglas M. McCarty,
Somanathan Suryanarayan,
Laurence O. Whiteley
Publication year - 2021
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

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