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 …
Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood–brain barrier gene delivery
H Fu, J DiRosario, S Killedar, K Zaraspe, DM McCarty - Molecular Therapy, 2011 - cell.com
The greatest challenge in developing therapies for mucopolysaccharidosis (MPS) IIIB is to
achieve efficient central nervous system (CNS) delivery across the blood–brain barrier …
achieve efficient central nervous system (CNS) delivery across the blood–brain barrier …
Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice
FJ Duncan, BJ Naughton, K Zaraspe, DA Murrey… - Molecular Therapy, 2015 - cell.com
Mucopolysaccharidosis (MPS) IIIA is a neuropathic lysosomal storage disease caused by
deficiency in N-sulfoglucosamine sulfohydrolase (SGSH). Genome-wide gene expression …
deficiency in N-sulfoglucosamine sulfohydrolase (SGSH). Genome-wide gene expression …
Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10
JA Gilkes, MD Bloom, CD Heldermon - Gene therapy, 2016 - nature.com
Sanfilippo syndrome type B (mucopolysaccharidosis IIIB, MPS IIIB) is a lysosomal storage
disease resulting from deficiency of N-acetyl-glucosaminidase (NAGLU) activity. To …
disease resulting from deficiency of N-acetyl-glucosaminidase (NAGLU) activity. To …
Toward a gene therapy for neurological and somatic MPSIIIA
V Haurigot, F Bosch - Rare Diseases, 2013 - Taylor & Francis
Mucopolysaccharidosis Type IIIA (MPSIIIA) represents an unmet medical need. MPSIIIA
shares with many other lysosomal storage disorders (LSD) the characteristic of being a …
shares with many other lysosomal storage disorders (LSD) the characteristic of being a …
AAVrh10 vector corrects disease pathology in MPS IIIA mice and achieves widespread distribution of SGSH in large animal brains
M Hocquemiller, KM Hemsley, ML Douglass… - … Therapy-Methods & …, 2020 - cell.com
Patients with mucopolysaccharidosis type IIIA (MPS IIIA) lack the lysosomal enzyme
sulfamidase (SGSH), which is responsible for the degradation of heparan sulfate (HS). Build …
sulfamidase (SGSH), which is responsible for the degradation of heparan sulfate (HS). Build …
Comparative effectiveness of intracerebroventricular, intrathecal, and intranasal routes of AAV9 vector administration for genetic therapy of neurologic disease in …
LR Belur, M Romero, J Lee… - Frontiers in Molecular …, 2021 - frontiersin.org
Mucopolysaccharidosis type I (MPS I) is an inherited metabolic disorder caused by
deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). The two current treatments …
deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). The two current treatments …
Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID
C Roca, S Motas, S Marcó, A Ribera… - Human Molecular …, 2017 - academic.oup.com
Gene therapy is a promising therapeutic alternative for Lysosomal Storage Disorders (LSD),
as it is not necessary to correct the genetic defect in all cells of an organ to achieve …
as it is not necessary to correct the genetic defect in all cells of an organ to achieve …
[HTML][HTML] Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: trial in progress-NCT04201405
JL Kinsella, RF Wynn, B Bigger, AJ Thrasher, C Booth… - Blood, 2020 - Elsevier
Autologous ex vivo hematopoietic stem cell gene therapy is particularly relevant in
lysosomal storage diseases (LSD) as it offers the prospect of both a safe transplant, as …
lysosomal storage diseases (LSD) as it offers the prospect of both a safe transplant, as …
Generation of a novel immunodeficient mouse model of Mucopolysaccharidosis type IIIA to test human stem cell-based therapies
O Mandolfo, H Parker, YI Learmonth, RJ Holley… - Molecular Genetics and …, 2024 - Elsevier
Abstract Mucopolysaccharidosis Type IIIA (MPSIIIA) is a rare inherited lysosomal storage
disease caused by mutations in the SGSH gene. This genetic variation results in the …
disease caused by mutations in the SGSH gene. This genetic variation results in the …