Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution

Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic diseases. In the present study, we first investig...

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Veröffentlicht in:	Molecular therapy. Methods & clinical development Jg. 28; S. 220 - 237
Hauptverfasser:	Cabanes-Creus, Marti, Navarro, Renina Gale, Liao, Sophia H.Y., Scott, Suzanne, Carlessi, Rodrigo, Roca-Pinilla, Ramon, Knight, Maddison, Baltazar, Grober, Zhu, Erhua, Jones, Matthew, Denisenko, Elena, Forrest, Alistair R.R., Alexander, Ian E., Tirnitz-Parker, Janina E.E., Lisowski, Leszek
Format:	Journal Article
Sprache:	Englisch
Veröffentlicht:	United States Elsevier Inc 09.03.2023 American Society of Gene & Cell Therapy Elsevier
Schlagworte:	adeno-associated vector gene therapy humanized FRG liver lobule liver zonation lobular transduction Original single-nucleus RNA sequencing liver lobule liver zonation single-nucleus RNA sequencing gene therapy lobular transduction humanized FRG adeno-associated vector
ISSN:	2329-0501, 2329-0501
Online-Zugang:	Volltext
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