[HTML][HTML] Ex vivo gene therapy treats bone complications of mucopolysaccharidosis type II mouse models through bone remodeling reactivation
M Wada, Y Shimada, S Iizuka, N Ishii, H Hiraki… - … Therapy-Methods & …, 2020 - cell.com
Mucopolysaccharidosis type II is a disease caused by organ accumulation of
glycosaminoglycans due to iduronate 2-sulfatase deficiency. This study investigated the …
glycosaminoglycans due to iduronate 2-sulfatase deficiency. This study investigated the …
Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term …
M Kosuga, S Takahashi, K Sasaki, XK Li, M Fujino… - Molecular Therapy, 2000 - cell.com
Recombinant adenoviruses expressing human b-glucuronidase (AxCAhGUS) and CTLA-4Ig
(AxCACTLA-4Ig) were generated and therapeutic efficacy was investigated using a murine …
(AxCACTLA-4Ig) were generated and therapeutic efficacy was investigated using a murine …
Enzymatic correction and cross-correction of mucopolysaccharidosis type I fibroblasts by adeno-associated virus-mediated transduction of the alpha-L-iduronidase …
SD Hartung, RG Reddy, CB Whitley… - Human gene therapy, 1999 - liebertpub.com
Mucopolysaccharidosis type I (MPS I), a deficiency in the lysosomal enzyme alpha-L-
iduronidase (IDUA), is characterized by skeletal abnormalities, hepatosplenomegaly and …
iduronidase (IDUA), is characterized by skeletal abnormalities, hepatosplenomegaly and …
Delivery of an adeno-associated virus vector into cerebrospinal fluid attenuates central nervous system disease in mucopolysaccharidosis type II mice
C Hinderer, N Katz, JP Louboutin, P Bell, H Yu… - Human Gene …, 2016 - liebertpub.com
Mucopolysaccharidosis type II (MPS II) is a rare X-linked genetic disorder caused by
deficiency of the lysosomal enzyme iduronate-2-sulfatase (IDS), leading to impaired …
deficiency of the lysosomal enzyme iduronate-2-sulfatase (IDS), leading to impaired …
Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human α-L …
SD Hartung, JL Frandsen, D Pan, BL Koniar… - Molecular Therapy, 2004 - cell.com
Murine models of lysosomal storage diseases provide an opportunity to evaluate the
potential for gene therapy to prevent systemic manifestations of the disease. To determine …
potential for gene therapy to prevent systemic manifestations of the disease. To determine …
An improved adeno-associated virus vector for neurological correction of the mouse model of mucopolysaccharidosis IIIA
Patients with the lysosomal storage disease mucopolysaccharidosis IIIA (MPSIIIA) lack the
lysosomal enzyme N-sulfoglucosamine sulfohydrolase (SGSH), one of the many enzymes …
lysosomal enzyme N-sulfoglucosamine sulfohydrolase (SGSH), one of the many enzymes …
Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer
TM Daly, KK Ohlemiller, MS Roberts, CA Vogler… - Gene therapy, 2001 - nature.com
For many inborn errors of metabolism, early treatment is critical to prevent long-term
developmental sequelae. We have previously shown that systemic treatment of neonatal …
developmental sequelae. We have previously shown that systemic treatment of neonatal …
Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector
CJ Rouse, K Hawkins, N Kabbej… - Human Molecular …, 2023 - academic.oup.com
Abstract Mucopolysaccharidosis type IIIB (MPS IIIB) is an autosomal recessive lysosomal
storage disease caused by mutations in the gene that encodes the protein N-acetyl …
storage disease caused by mutations in the gene that encodes the protein N-acetyl …
Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice
H Fu, L Kang, JS Jennings, SS Moy, A Perez… - Gene therapy, 2007 - nature.com
Mucopolysaccharidosis (MPS) IIIB is an inherited lysosomal storage disease, caused by the
deficiency of α-N-acetylglucosaminidase (NaGlu), resulting in severe global neurological …
deficiency of α-N-acetylglucosaminidase (NaGlu), resulting in severe global neurological …
Intra-articular nonviral gene therapy in mucopolysaccharidosis I mice
Abstract Mucopolysaccharidosis type I (MPS I) is caused by the lysosomal accumulation of
glycosaminoglycans (GAGs) due to the deficiency of the enzyme alpha-L-iduronidase …
glycosaminoglycans (GAGs) due to the deficiency of the enzyme alpha-L-iduronidase …