IIIB 型粘多糖病: AAV 疗法的现状回顾和探索
CJ Rouse, VN Jensen, CD Heldermon - 中国神经再生研究(英文版), 2024 - sjzsyj.com.cn
Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene
that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan …
that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan …
Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape
CJ Rouse, VN Jensen… - Neural Regeneration …, 2023 - journals.lww.com
Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene
that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan …
that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan …
Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery
H Fu, RJ Samulski, TJ McCown, YJ Picornell… - Molecular Therapy, 2002 - cell.com
Mucopolysaccharidosis (MPS) IIIB is characterized by mild somatic features and severe
neurological diseases leading to premature death. No definite treatment is available for MPS …
neurological diseases leading to premature death. No definite treatment is available for MPS …
Restoration of central nervous system α‐N‐acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal …
H Fu, J DiRosario, L Kang, J Muenzer… - The journal of gene …, 2010 - Wiley Online Library
Background Finding efficient central nervous system (CNS) delivery approaches has been
the major challenge facing therapeutic development for treating diseases with global …
the major challenge facing therapeutic development for treating diseases with global …
Intracranial gene delivery of LV‐NAGLU vector corrects neuropathology in murine MPS IIIB
C Di Domenico, GRD Villani… - American journal of …, 2009 - Wiley Online Library
Mucopolysacccharidosis (MPS) IIIB is an inherited lysosomal storage disorder caused by the
deficiency of alpha‐N‐acetylglucosaminidase (NAGLU). The disease is characterized by …
deficiency of alpha‐N‐acetylglucosaminidase (NAGLU). The disease is characterized by …
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
CD Heldermon, EY Qin, KK Ohlemiller, ED Herzog… - Gene therapy, 2013 - nature.com
Abstract Mucopolysaccharidosis type IIIB (MPS IIIB) or Sanfilippo Syndrome type B is a
lysosomal storage disease resulting from the deficiency of N-acetyl glucosaminidase …
lysosomal storage disease resulting from the deficiency of N-acetyl glucosaminidase …
Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I
DA Wolf, AW Lenander, Z Nan, LR Belur… - Neurobiology of …, 2011 - Elsevier
The mucopolysaccharidoses (MPSs) are a group of 11 storage diseases caused by
disruptions in glycosaminoglycan (GAG) catabolism, leading to their accumulation in …
disruptions in glycosaminoglycan (GAG) catabolism, leading to their accumulation in …
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 …
[引用][C] Development and validation of a novel adeno-associated viral gene therapy for mucopolysaccharidosis type IIIB (MPS IIIB)
N Ballout, L Verdu, C Santiago, M Rouahi… - Molecular Genetics and …, 2023 - Elsevier
[引用][C] Development and validation of a novel AAV gene therapy for mucopolysaccharidosis type IIIB in large animal
EG Banchi, R Alonso, S Jacquot, K Fransquin… - Molecular Genetics and …, 2024 - Elsevier