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Publications-Presentations-Press

PHOENIX NEST BIOTECH NEWS & EVENTS

Publications

Publications relevant to Phoenix Nest programs; authored, funded, or in collaboration with employees of Phoenix Nest and or Jonah’s Just Begun-Foundation to Cure Sanfilippo.

Pan X, Taherzadeh M, Bose P, Heon-Roberts R, Nguyen ALA, Xu T, Pará C, Yamanaka Y, Priestman DA, Platt FM, Khan S, Fnu N, Tomatsu S, Morales CR, Pshezhetsky AV. Glucosamine amends CNS pathology in mucopolysaccharidosis IIIC mouse expressing misfolded HGSNAT. J Exp Med. 2022 Aug 1;219(8):e20211860. doi: 10.1084/jem.20211860. Epub 2022 Jun 15. PMID: 35704026; PMCID: PMC9204472.

Ludwig J, Sawant OB, Wood J, Singamsetty S, Pan X, Bonilha VL, Rao S, Pshezhetsky AV. Histological characterization of retinal degeneration in mucopolysaccharidosis type IIIC. Exp Eye Res. 2023 Apr;229:109433. doi: 10.1016/j.exer.2023.109433. Epub 2023 Feb 27. PMID: 36858249; PMCID: PMC10103010.

Viana GM, Priestman DA, Platt FM, Khan S, Tomatsu S, Pshezhetsky AV. Brain Pathology in Mucopolysaccharidoses (MPS) Patients with Neurological Forms. J Clin Med. 2020 Feb 1;9(2):396. doi: 10.3390/jcm9020396. PMID: 32024172; PMCID: PMC7073982.

Cheng KW, Wang F, Lopez GA, Singamsetty S, Wood J, Dickson PI, Chou TF. Evaluation of artificial signal peptides for secretion of two lysosomal enzymes in CHO cells. Biochem J. 2021 Jun 25;478(12):2309-2319. doi: 10.1042/BCJ20210015. PMID: 34032266.

 

Wang F, et al. (2020). Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice.  Mol Pharm. 2021 Jan 4;18(1):214-227. doi: 10.1021/acs.molpharmaceut.0c00831. Epub 2020 Dec 15. PMID: 33320673

 

Heon-Roberts R, Nguyen ALA, Pshezhetsky AV. Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo Disease.  J Clin Med. 2020 Jan 27;9(2):344. doi: 10.3390/jcm9020344. PMID: 32012694

 

Martins C, et al. (2019). Molecular characterization of a large group of Mucopolysaccharidosis type IIIC patients reveals the evolutionary history of the disease.  Hum Mutat. 2019 Aug;40(8):1084-1100. doi: 10.1002/humu.23752. Epub 2019 Jun 22. PMID: 31228227

 

Carla Martins, et al. (2015) Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model.  Brain 2015 Feb;138(Pt 2):336-55. doi: 10.1093/brain/awu355. Epub 2015 Jan 6. PMID: 25567323

Presentations & Press

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