Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I

Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I

Mucopolysaccharidosis type I (MPS I) is a rare metabolic disorder caused by deficiency of α-L-iduronidase (IDUA), resulting in glycosaminoglycan (GAG) accumulation and multisystemic disease. Current treatments include hematopoietic stem cell transplantation and enzyme replacement therapy, but these...

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Main Authors: Lalitha R. Belur, Avery K. Huber, Hillary Mantone, Mason Robertson, Miles C. Smith, Andrea D. Karlen, Kelley F. Kitto, Li Ou, Chester B. Whitley, Elizabeth Braunlin, Justin Furcich, Troy C. Lund, Davis Seelig, Carolyn A. Fairbanks, Nicholas Buss, Kwi Hye Kim, R. Scott McIvor
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
MPS I
gene therapy
AAV9
intrathecal
intravenous
dose response
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050124001852
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