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Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength.
Martin PT, Zygmunt DA, Ashbrook A, Hamilton S, Packer D, Birch SM, Bettis AK, Balog-Alvarez CJ, Guo LJ, Nghiem PP, Kornegay JN. Martin PT, et al. PLoS One. 2021 Mar 26;16(3):e0248721. doi: 10.1371/journal.pone.0248721. eCollection 2021. PLoS One. 2021. PMID: 33770101 Free PMC article.
Serum chemistry, hematology, and cardiac function measures were largely unchanged by treatment. Cumulatively, these data show that short-term intravenous treatment of GRMD dogs with rAAVrh74.MHCK7.GALGT2 at high doses can induce muscle glycosylation and utrophin expression …
Serum chemistry, hematology, and cardiac function measures were largely unchanged by treatment. Cumulatively, these data show that short- …
Expansion of B4GALT7 linkeropathy phenotype to include perinatal lethal skeletal dysplasia.
Mihalic Mosher T, Zygmunt DA, Koboldt DC, Kelly BJ, Johnson LR, McKenna DS, Hood BC, Hickey SE, White P, Wilson RK, Martin PT, McBride KL. Mihalic Mosher T, et al. Among authors: martin pt. Eur J Hum Genet. 2019 Oct;27(10):1569-1577. doi: 10.1038/s41431-019-0464-8. Epub 2019 Jul 5. Eur J Hum Genet. 2019. PMID: 31278392 Free PMC article.
We report a family with three affected pregnancies showing short limbs, cystic hygroma, and perinatal death. Two spontaneously aborted; one survived 1 day after term delivery, and had short limbs, bell-shaped thorax, 11 ribs, absent thumbs, and cleft palate. ...
We report a family with three affected pregnancies showing short limbs, cystic hygroma, and perinatal death. Two spontaneously aborted; one …
Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone.
Cramer ML, Shao G, Rodino-Klapac LR, Chicoine LG, Martin PT. Cramer ML, et al. Among authors: martin pt. Hum Gene Ther. 2017 Jun;28(6):493-509. doi: 10.1089/hum.2016.113. Epub 2017 Mar 23. Hum Gene Ther. 2017. PMID: 28345428 Free PMC article.
These data demonstrate that prednisone can reduce the extent of intramuscular T-cell infiltrates in AAV-treated muscles, which may aid in achieving long-term transgene expression, as may the induction of PDL2 expression on skeletal myofibers to promote PD1-mediated program …
These data demonstrate that prednisone can reduce the extent of intramuscular T-cell infiltrates in AAV-treated muscles, which may aid in ac …
Glycobiology of neuromuscular disorders.
Martin PT, Freeze HH. Martin PT, et al. Glycobiology. 2003 Aug;13(8):67R-75R. doi: 10.1093/glycob/cwg077. Epub 2003 May 7. Glycobiology. 2003. PMID: 12736200 Review.
In addition, we review evidence that glycobiology is important in other disorders that affect muscle, including hereditary inclusion body myopathy type II and congenital disorders of glycosylation. Finally, we discuss the long-term potential of glycotherapies for muscle di …
In addition, we review evidence that glycobiology is important in other disorders that affect muscle, including hereditary inclusion body my …