Kakkis E - Search Results

1

Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression.

Wang L, Warzecha CC, Kistner A, Chichester JA, Bell P, Buza EL, He Z, Pampena MB, Couthouis J, Sethi S, McKeever K, Betts MR, Kakkis E, Wilson JM, Wadsworth S, Sullivan BA. Wang L, et al. Among authors: kakkis e. Mol Ther Methods Clin Dev. 2022 Jan 19;24:292-305. doi: 10.1016/j.omtm.2022.01.007. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2022. PMID: 35211641 Free PMC article.

2

Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept.

Cadaoas J, Hu H, Boyle G, Gomero E, Mosca R, Jayashankar K, Machado M, Cullen S, Guzman B, van de Vlekkert D, Annunziata I, Vellard M, Kakkis E, Koppaka V, d'Azzo A. Cadaoas J, et al. Among authors: kakkis e. Mol Ther Methods Clin Dev. 2020 Nov 20;20:191-203. doi: 10.1016/j.omtm.2020.11.012. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33426146 Free PMC article.

3

Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment--A retrospective chart review.

Vockley J, Marsden D, McCracken E, DeWard S, Barone A, Hsu K, Kakkis E. Vockley J, et al. Among authors: kakkis e. Mol Genet Metab. 2015 Sep-Oct;116(1-2):53-60. doi: 10.1016/j.ymgme.2015.06.006. Epub 2015 Jun 18. Mol Genet Metab. 2015. PMID: 26116311 Free PMC article. Review.

4

Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses.

Jones SA, Marsden D, Koutsoukos T, Sniadecki J, Tylee K, Phillippo S, Kakkis E. Jones SA, et al. Among authors: kakkis e. Mol Genet Metab. 2020 Aug;130(4):255-261. doi: 10.1016/j.ymgme.2020.06.004. Epub 2020 Jun 11. Mol Genet Metab. 2020. PMID: 32563631 Free article.

5

Conventional and Unconventional Therapeutic Strategies for Sialidosis Type I.

Mosca R, van de Vlekkert D, Campos Y, Fremuth LE, Cadaoas J, Koppaka V, Kakkis E, Tifft C, Toro C, Allievi S, Gellera C, Canafoglia L, Visser G, Annunziata I, d'Azzo A. Mosca R, et al. Among authors: kakkis e. J Clin Med. 2020 Mar 4;9(3):695. doi: 10.3390/jcm9030695. J Clin Med. 2020. PMID: 32143456 Free PMC article.

6

Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII.

Cadaoas J, Boyle G, Jungles S, Cullen S, Vellard M, Grubb JH, Jurecka A, Sly W, Kakkis E. Cadaoas J, et al. Among authors: kakkis e. Mol Genet Metab. 2020 May;130(1):65-76. doi: 10.1016/j.ymgme.2020.02.009. Epub 2020 Mar 6. Mol Genet Metab. 2020. PMID: 32192868 Free article. Clinical Trial.

7

Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice.

Dvorak-Ewell M, Wendt D, Hague C, Christianson T, Koppaka V, Crippen D, Kakkis E, Vellard M. Dvorak-Ewell M, et al. Among authors: kakkis e. PLoS One. 2010 Aug 16;5(8):e12194. doi: 10.1371/journal.pone.0012194. PLoS One. 2010. PMID: 20808938 Free PMC article.

8

Substantial deficiency of free sialic acid in muscles of patients with GNE myopathy and in a mouse model.

Chan YM, Lee P, Jungles S, Morris G, Cadaoas J, Skrinar A, Vellard M, Kakkis E. Chan YM, et al. Among authors: kakkis e. PLoS One. 2017 Mar 7;12(3):e0173261. doi: 10.1371/journal.pone.0173261. eCollection 2017. PLoS One. 2017. PMID: 28267778 Free PMC article.

9

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice.

Watson G, Bastacky J, Belichenko P, Buddhikot M, Jungles S, Vellard M, Mobley WC, Kakkis E. Watson G, et al. Among authors: kakkis e. Gene Ther. 2006 Jun;13(11):917-25. doi: 10.1038/sj.gt.3302735. Gene Ther. 2006. PMID: 16482204

10

First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient.

Fox JE, Volpe L, Bullaro J, Kakkis ED, Sly WS. Fox JE, et al. Among authors: kakkis ed. Mol Genet Metab. 2015 Feb;114(2):203-8. doi: 10.1016/j.ymgme.2014.10.017. Epub 2014 Nov 7. Mol Genet Metab. 2015. PMID: 25468648 Free PMC article.

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