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Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives
Lysosomes and lysosomal enzymes play a central role in numerous cellular processes,
including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies …
including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies …
Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment: a special review
Mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A, is a rare, autosomal
recessive disorder caused by a deficiency of the lysosomal enzyme N-acetylgalatosamine-6 …
recessive disorder caused by a deficiency of the lysosomal enzyme N-acetylgalatosamine-6 …
International Morquio A Registry: clinical manifestation and natural course of Morquio A disease
Summary Mucopolysaccharidosis IVA (MPS IVA; Morquio A disease) is a lysosomal storage
disorder caused by deficiency of N‐acetylgalactosamine‐6‐sulfate sulfatase. The natural …
disorder caused by deficiency of N‐acetylgalactosamine‐6‐sulfate sulfatase. The natural …
Mucopolysaccharidosis IVA and glycosaminoglycans
Abstract Mucopolysaccharidosis IVA (MPS IVA; Morquio A: OMIM 253000) is a lysosomal
storage disease with an autosomal recessive trait caused by the deficiency of N …
storage disease with an autosomal recessive trait caused by the deficiency of N …
Morquio A syndrome: diagnosis and current and future therapies
S Tomatsu, E Yasuda, P Patel… - Pediatric …, 2014 - pmc.ncbi.nlm.nih.gov
Morquio A syndrome is an autosomal recessive disorder, one of 50 lysosomal storage
diseases (LSDs), and is caused by the deficiency of N-acetylgalactosamine-6-sulfate …
diseases (LSDs), and is caused by the deficiency of N-acetylgalactosamine-6-sulfate …
Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
J Bertolin, V Sánchez, A Ribera, ML Jaén… - Nature …, 2021 - nature.com
Mucopolysaccharidosis type IVA (MPSIVA) or Morquio A disease, a lysosomal storage
disorder, is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency …
disorder, is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency …
Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome
Mucopolysaccharidosis IVA (MPS IVA, Morquio A disease) is an inherited lysosomal storage
disorder that features skeletal chondrodysplasia caused by deficiency of N …
disorder that features skeletal chondrodysplasia caused by deficiency of N …
Morquio A Syndrome‐Associated Mutations: A Review of Alterations in the GALNS Gene and a New Locus‐Specific Database
A Morrone, A Caciotti, R Atwood, K Davidson… - Human …, 2014 - Wiley Online Library
Morquio A syndrome (mucopolysaccharidosis IVA) is an autosomal recessive disorder that
results from deficient activity of the enzyme N‐acetylgalactosamine‐6‐sulfatase (GALNS) …
results from deficient activity of the enzyme N‐acetylgalactosamine‐6‐sulfatase (GALNS) …
Enzyme replacement therapy in a murine model of Morquio A syndrome
Abstract Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disorder caused
by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), leading to …
by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), leading to …
Pathogenesis of Morquio A syndrome: an autopsied case reveals systemic storage disorder
E Yasuda, K Fushimi, Y Suzuki, K Shimizu… - Molecular Genetics and …, 2013 - Elsevier
Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is a lysosomal storage disorder
caused by deficiency of N-acetylgalactosamine-6-sulfate sulfatase, which results in systemic …
caused by deficiency of N-acetylgalactosamine-6-sulfate sulfatase, which results in systemic …