Intravenous Delivery of a Gene Therapy Vector that Expresses an Antitransferrin Receptor 1 Nanobody-I2S Fusion Protein Rescued Central Nervous System Lysosomal Burden in Mucopolysaccharidosis II Mice.
Ruangsiriluk W, Jacobo SMP, Iskenderian A, Modi M, Chen N, Felice B, Yuan S, Mukherji ST, Rivera Hernandez M, Sunkara S, MacDonald A, Rajarshi G, Palmieri K, Crooker R, Wu R, Dalrymple M, Chheda M, Megahid D, Arce C, Shen M, Dasari B, Zhao X, Yalavarthi M, Rompikuntal P, Ayyadurai S, Fang J, Hozanovic A, Pescatore B, Bassous D, Romashko A, Galbreath E, Maccani A, Steinkellner J, Ehmann DE, Choi VW, Islam R.
Ruangsiriluk W, et al. Among authors: ehmann de.
Hum Gene Ther. 2025 Dec 16. doi: 10.1177/10430342251406283. Online ahead of print.
Hum Gene Ther. 2025.
PMID: 41457681