Przybilla MJ - Search Results

1

Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis.

Przybilla MJ, Stewart C, Carlson TW, Ou L, Koniar BL, Sidhu R, Kell PJ, Jiang X, Jarnes JR, O'Sullivan MG, Whitley CB. Przybilla MJ, et al. Mol Genet Metab Rep. 2021 Mar 25;27:100748. doi: 10.1016/j.ymgmr.2021.100748. eCollection 2021 Jun. Mol Genet Metab Rep. 2021. PMID: 33854948 Free PMC article.

2

Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I.

Ou L, Przybilla MJ, Koniar BL, Whitley CB. Ou L, et al. Among authors: przybilla mj. Mol Genet Metab Rep. 2016 Aug 13;8:87-93. doi: 10.1016/j.ymgmr.2015.11.004. eCollection 2016 Sep. Mol Genet Metab Rep. 2016. PMID: 27556013 Free PMC article.

3

RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system.

Ou L, Przybilla MJ, Koniar B, Whitley CB. Ou L, et al. Among authors: przybilla mj. Mol Genet Metab. 2018 Feb;123(2):105-111. doi: 10.1016/j.ymgme.2017.11.013. Epub 2017 Nov 28. Mol Genet Metab. 2018. PMID: 29198892 Free PMC article.

4

Metabolomics profiling reveals profound metabolic impairments in mice and patients with Sandhoff disease.

Ou L, Przybilla MJ, Whitley CB. Ou L, et al. Among authors: przybilla mj. Mol Genet Metab. 2019 Feb;126(2):151-156. doi: 10.1016/j.ymgme.2018.09.005. Epub 2018 Sep 14. Mol Genet Metab. 2019. PMID: 30236619 Free PMC article.

5

Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type B.

Przybilla MJ, Ou L, Tăbăran AF, Jiang X, Sidhu R, Kell PJ, Ory DS, O'Sullivan MG, Whitley CB. Przybilla MJ, et al. Mol Genet Metab. 2019 Feb;126(2):139-150. doi: 10.1016/j.ymgme.2018.11.002. Epub 2018 Nov 22. Mol Genet Metab. 2019. PMID: 30528226 Free article.

6

Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system.

Kim S, Przybilla MJ, Whitley CB, Ou L, Al-Kofahi M, Jarnes JR. Kim S, et al. Among authors: przybilla mj. Mol Genet Metab Rep. 2022 Sep 18;33:100917. doi: 10.1016/j.ymgmr.2022.100917. eCollection 2022 Dec. Mol Genet Metab Rep. 2022. PMID: 36159322 Free PMC article.

7

SAAMP 2.0: An algorithm to predict genotype-phenotype correlation of lysosomal storage diseases.

Ou L, Przybilla MJ, Whitley CB. Ou L, et al. Among authors: przybilla mj. Clin Genet. 2018 May;93(5):1008-1014. doi: 10.1111/cge.13226. Epub 2018 Mar 5. Clin Genet. 2018. PMID: 29396849 Free PMC article.

8

Proteomic analysis of mucopolysaccharidosis I mouse brain with two-dimensional polyacrylamide gel electrophoresis.

Ou L, Przybilla MJ, Whitley CB. Ou L, et al. Among authors: przybilla mj. Mol Genet Metab. 2017 Jan-Feb;120(1-2):101-110. doi: 10.1016/j.ymgme.2016.10.001. Epub 2016 Oct 11. Mol Genet Metab. 2017. PMID: 27742266 Free PMC article.

9

A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases.

Ou L, Przybilla MJ, Tăbăran AF, Overn P, O'Sullivan MG, Jiang X, Sidhu R, Kell PJ, Ory DS, Whitley CB. Ou L, et al. Among authors: przybilla mj. Gene Ther. 2020 May;27(5):226-236. doi: 10.1038/s41434-019-0120-5. Epub 2020 Jan 2. Gene Ther. 2020. PMID: 31896760 Free PMC article.

10

A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I.

Ou L, Przybilla MJ, Ahlat O, Kim S, Overn P, Jarnes J, O'Sullivan MG, Whitley CB. Ou L, et al. Among authors: przybilla mj. Mol Ther. 2020 Jun 3;28(6):1442-1454. doi: 10.1016/j.ymthe.2020.03.018. Epub 2020 Apr 8. Mol Ther. 2020. PMID: 32278382 Free PMC article.

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