Mucopolysaccharidosis type II: one hundred years of research, diagnosis, and treatment
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) was first described by Dr. Charles
Hunter in 1917. Since then, about one hundred years have passed and Hunter syndrome …
Hunter in 1917. Since then, about one hundred years have passed and Hunter syndrome …
Ready for repair? Gene editing enters the clinic for the treatment of human disease
MPT Ernst, M Broeders, P Herrero-Hernandez… - … Therapy Methods & …, 2020 - cell.com
We present an overview of clinical trials involving gene editing using clustered interspaced
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …
Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice
Most lysosomal storage diseases (LSDs) involve progressive central nervous system (CNS)
impairment, resulting from deficiency of a lysosomal enzyme. Treatment of neuronopathic …
impairment, resulting from deficiency of a lysosomal enzyme. Treatment of neuronopathic …
Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders
Lysosomes play a central role in cellular homeostasis and alterations in this compartment
associate with many diseases. The most studied example is that of lysosomal storage …
associate with many diseases. The most studied example is that of lysosomal storage …
Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms
HFE Gleitz, AY Liao, JR Cook, SF Rowlston… - EMBO molecular …, 2018 - embopress.org
The pediatric lysosomal storage disorder mucopolysaccharidosis type II is caused by
mutations in IDS, resulting in accumulation of heparan and dermatan sulfate, causing severe …
mutations in IDS, resulting in accumulation of heparan and dermatan sulfate, causing severe …
Gene therapy for Mucopolysaccharidoses
K Sawamoto, HH Chen, CJ Alméciga-Díaz… - Molecular genetics and …, 2018 - Elsevier
Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders (LSDs) caused
by a deficiency of lysosomal enzymes, leading to a wide range of various clinical symptoms …
by a deficiency of lysosomal enzymes, leading to a wide range of various clinical symptoms …
Delivering gene therapy for mucopolysaccharide diseases
SR Wood, BW Bigger - Frontiers in Molecular Biosciences, 2022 - frontiersin.org
Mucopolysaccharide diseases are a group of paediatric inherited lysosomal storage
diseases that are caused by enzyme deficiencies, leading to a build-up of …
diseases that are caused by enzyme deficiencies, leading to a build-up of …
Differences in MPS I and MPS II disease manifestations
CS Hampe, BD Yund, PJ Orchard, TC Lund… - International journal of …, 2021 - mdpi.com
Mucopolysaccharidosis (MPS) type I and II are two closely related lysosomal storage
diseases associated with disrupted glycosaminoglycan catabolism. In MPS II, the first step of …
diseases associated with disrupted glycosaminoglycan catabolism. In MPS II, the first step of …
[HTML][HTML] Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
S Marcó, V Haurigot, ML Jaén, A Ribera… - … Therapy Methods & …, 2021 - cell.com
Delivery of adeno-associated viral vectors (AAVs) to cerebrospinal fluid (CSF) has emerged
as a promising approach to achieve widespread transduction of the central nervous system …
as a promising approach to achieve widespread transduction of the central nervous system …
Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells
MC Smith, LR Belur, AD Karlen, O Erlanson… - Human gene …, 2022 - liebertpub.com
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is an X-linked recessive lysosomal
disease caused by deficiency of iduronate-2-sulfatase (IDS). The absence of IDS results in …
disease caused by deficiency of iduronate-2-sulfatase (IDS). The absence of IDS results in …