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Transcriptomicstranscriptional profiling of brain from WT, AAV9-Ids or Null-treated MPSII mice.
The experiment evaluates the therapeutic effect brain injected AAV9-IDS (Adeno-associated virus 9 encoding IDS enzyme) treatment in a mouse model of Mucopolysaccharidosis Type II (MPSII). Microarrays were performed in order to compare the transcriptional profiling after the treatment. Three groups w...
ORGANISM(S): Mus musculus 
2019-05-20 | E-MTAB-4728 | biostudies-arrayexpress
UnknownGlial degeneration with oxidative damage drives neuronal demise in MPSII disease.
Not available
S-EPMC5108318 | biostudies-literature
UnknownIDS crossing of the blood-brain barrier corrects CNS defects in MPSII mice.
Not available
S-EPMC2725243 | biostudies-literature
UnknownSustained long-term disease correction in a murine model of MPSII following stem cell gene therapy.
Not available
S-EPMC10618237 | biostudies-literature
UnknownTargeting Brain Disease in MPSII: Preclinical Evaluation of IDS-Loaded PLGA Nanoparticles.
Not available
S-EPMC6514713 | biostudies-literature
UnknownDesign and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.
Not available
S-EPMC11214401 | biostudies-literature
UnknownCNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome).
Not available
S-EPMC5033872 | biostudies-literature
UnknownA Rapid Two-Step Iduronate-2-Sulfatatse Enzymatic Activity Assay for MPSII Pharmacokinetic Assessment.
Not available
S-EPMC5874215 | biostudies-literature
UnknownSevere tracheal and bronchial collapse in adults with type II mucopolysaccharidosis.
Not available
S-EPMC4845328 | biostudies-literature
UnknownDiverse clinical outcome of Hunter syndrome in patients with chromosomal aberration encompassing entire and partial IDS deletions: what is important for early diagnosis and counseling?
Not available
S-EPMC8868176 | biostudies-literature
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