Targeting the root cause of mucopolysaccharidosis IIIA with a new scAAV9 gene replacement vector
TA Bobo, PN Samowitz, MI Robinson, H Fu - Molecular Therapy-Methods & …, 2020 - cell.com
No treatment is available to address the unmet needs of mucopolysaccharidosis (MPS) IIIA
patients. Targeting the root cause, we developed a new self-complementary adeno …
patients. Targeting the root cause, we developed a new self-complementary adeno …
Adeno-associated virus-based gene therapy delivering combinations of two growth-associated genes to MPS IVA mice
E Rintz, B Celik, N Fnu, AM Herreño-Pachón… - … Therapy-Nucleic Acids, 2024 - cell.com
Mucopolysaccharidosis type IVA (MPS IVA) is caused by a deficiency of the galactosamine
(N-acetyl)-6-sulfatase (GALNS) enzyme responsible for the degradation of specific …
(N-acetyl)-6-sulfatase (GALNS) enzyme responsible for the degradation of specific …
93. Long-Term CNS and Somatic Correction in MPS II Mice after Intrathecal Delivery of AAV 9 Vector
L Kang, J Muenzer, BC Eggert - 2007 - cell.com
Mucopolysaccharidosis II (MPS II) is an X-linked lysosomal storage disorder due to the
deficiency of the enzyme, iduronate sulfatase (Id-S). No treatment is available for the central …
deficiency of the enzyme, iduronate sulfatase (Id-S). No treatment is available for the central …
Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice …
Y Kamata, A Tanabe, A Kanaji, M Kosuga, Y Fukuhara… - Gene therapy, 2003 - nature.com
Systemic injection of an adenovirus vector into adult mice resulted in pathological
improvements in multiple visceral organs of mice with mucopolysaccharidosis VII; however …
improvements in multiple visceral organs of mice with mucopolysaccharidosis VII; however …
Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors
G Liu, I Martins, JA Wemmie, JA Chiorini… - Journal of …, 2005 - Soc Neuroscience
Lysosomal storage diseases (LSDs) represent a significant portion of inborn metabolic
disorders. More than 60% of LSDs have CNS involvement. LSD therapies for systemic …
disorders. More than 60% of LSDs have CNS involvement. LSD therapies for systemic …
Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum
A Cressant, N Desmaris, L Verot, T Bréjot… - Journal of …, 2004 - Soc Neuroscience
Sanfilippo syndrome is a mucopolysaccharidosis (MPS) caused by a lysosomal enzyme
defect interrupting the degradation pathway of heparan sulfates. Affected children develop …
defect interrupting the degradation pathway of heparan sulfates. Affected children develop …
In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon
S Marcó, V Haurigot, F Bosch - Human Gene Therapy, 2019 - liebertpub.com
For most lysosomal storage diseases (LSDs), there is no cure. Gene therapy is an attractive
tool for treatment of LSDs caused by deficiencies in secretable lysosomal enzymes, in which …
tool for treatment of LSDs caused by deficiencies in secretable lysosomal enzymes, in which …
[HTML][HTML] Study of the neurodegenerative process in Mucopolysaccharidosis type VII and its correction by gene therapy
A Onieva Salgado - 2023 - ddd.uab.cat
La mucopolisacaridosis tipus VII és un trastorn autosòmic recessiu causat per una
deficiència de β-glucuronidasa,(GUSB) que condueix a l'acumulació de glicosaminoglicans …
deficiència de β-glucuronidasa,(GUSB) que condueix a l'acumulació de glicosaminoglicans …
Mucopolysaccharidoses I and II: brief review of therapeutic options and supportive/palliative therapies
H Nan, C Park, S Maeng - BioMed Research International, 2020 - Wiley Online Library
Purpose. Mucopolysaccharidoses (MPS) are group of inherited lysosomal storage diseases
caused by mutations of enzymes involved in catalyzing different glycosaminoglycans …
caused by mutations of enzymes involved in catalyzing different glycosaminoglycans …