Buyankhishig Tsogtbaatar
- Lysosomal Storage Disorders Research
- Trypanosoma species research and implications
- Cellular transport and secretion
- Amyotrophic Lateral Sclerosis Research
- Glycogen Storage Diseases and Myoclonus
- Parkinson's Disease Mechanisms and Treatments
- Alzheimer's disease research and treatments
- Salmonella and Campylobacter epidemiology
- Glycosylation and Glycoproteins Research
- Prion Diseases and Protein Misfolding
- Viral gastroenteritis research and epidemiology
- Influenza Virus Research Studies
- Cerebrospinal fluid and hydrocephalus
- Biofuel production and bioconversion
- Neonatal and fetal brain pathology
- Polysaccharides and Plant Cell Walls
- Animal Virus Infections Studies
- Escherichia coli research studies
- Bacteriophages and microbial interactions
- Respiratory viral infections research
- Cancer Research and Treatments
Denali Therapeutics (United States)
2020-2024
Scripps Research Institute
2016-2019
Delivery of biotherapeutics across the blood-brain barrier (BBB) is a challenge. Many approaches fuse to platforms that bind transferrin receptor (TfR), brain endothelial cell target, facilitate receptor-mediated transcytosis BBB. Here, we characterized pharmacological behavior two distinct TfR-targeted fused iduronate 2-sulfatase (IDS), lysosomal enzyme deficient in mucopolysaccharidosis type II (MPS II), and compared relative exposures functional activities both mouse models. IDS...
Abstract Background Frontotemporal dementia (FTD) is the most common cause of early-onset with 10-20% cases caused by mutations in one three genes: GRN , C9orf72 or MAPT . To effectively develop therapeutics for FTD, identification and characterization biomarkers to understand disease pathogenesis evaluate impact specific therapeutic strategies on target biology as well underlying pathology are essential. Moreover, tracking longitudinal changes these throughout progression crucial discern...
Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder caused by deficiency of the iduronate-2-sulfatase (IDS) enzyme, resulting in cellular accumulation glycosaminoglycans (GAGs) throughout body. Treatment MPS remains considerable challenge as current enzyme replacement therapies do not adequately control many aspects disease, including skeletal and neurological manifestations. We developed an IDS transport vehicle (ETV:IDS) that engineered to bind transferrin receptor; this...
We recently developed a blood-brain barrier (BBB)-penetrating enzyme transport vehicle (ETV) fused to the lysosomal iduronate 2-sulfatase (ETV:IDS) and demonstrated its ability reduce glycosaminoglycan (GAG) accumulation in brains of mouse model mucopolysaccharidosis (MPS) II. To accurately quantify GAGs, we plate-based high-throughput enzymatic digestion assay coupled with liquid chromatography-tandem mass spectrometry (LC-MS/MS) simultaneously measure heparan sulfate dermatan derived...
Abstract Delivery of biotherapeutics across the blood-brain barrier (BBB) is a challenge. Many approaches fuse to platforms that bind transferrin receptor (TfR), brain endothelial cell target, facilitate receptor-mediated transcytosis BBB. Here, we characterized pharmacological behavior two distinct TfR-targeted fused iduronate 2-sulfatase (IDS), lysosomal enzyme deficient in mucopolysaccharidosis type II (MPS II), and compared relative exposures functional activities both mouse models. IDS...