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1 . Academic Journal
Dual FKRP/FST gene therapy normalizes ambulation, increases strength, decreases pathology, and amplifies gene expression in LGMDR9 mice
저자
by
Lam, Patricia
;
Zygmunt, Deborah A.
;
Ashbrook, Anna
;
Bennett, Macey
;
Vetter, Tatyana A.
, et al.
소스
In
Molecular Therapy
7 August 2024 32(8):2604-2623
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7 . Academic Journal
Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency
저자
by
Lam, Patricia
;
Ashbrook, Anna
;
Zygmunt, Deborah A.
;
Yan, Cong
;
Du, Hong
, et al.
소스
In
Molecular Therapy - Methods & Clinical Development
8 September 2022 26:413-426
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7 . Academic Journal
Serum Antibodies to N-Glycolylneuraminic Acid Are Elevated in Duchenne Muscular Dystrophy and Correlate with Increased Disease Pathology in Cmah−/−mdx Mice
저자
by
Martin, Paul T.
;
Kawanishi, Kunio
;
Ashbrook, Anna
;
Golden, Bethannie
;
Samraj, Annie
, et al.
소스
In
The American Journal of Pathology
August 2021 191(8):1474-1486
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7 . Periodical
Dual FKRP/FSTgene therapy normalizes ambulation, increases strength, decreases pathology, and amplifies gene expression in LGMDR9 mice
저자
by
Lam, Patricia
;
Zygmunt, Deborah A.
;
Ashbrook, Anna
;
Bennett, Macey
;
Vetter, Tatyana A.
, et al.
소스
Molecular Therapy; August 2024, Vol. 32 Issue: 8 p2604-2623, 20p
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7 . Academic Journal
Development of Assays to Measure GNE Gene Potency and Gene Replacement in Skeletal Muscle.
저자
by
Zygmunt, Deborah A.
;
Lam, Patricia
;
Ashbrook, Anna
;
Koczwara, Katherine
;
Lek, Angela
, et al.
소스
Journal of Neuromuscular Diseases; 2023, Vol. 10 Issue 5, p797-812, 16p
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7 . Periodical
Development of Assays to Measure GNEGene Potency and Gene Replacement in Skeletal Muscle
저자
by
Zygmunt, Deborah A.
;
Lam, Patricia
;
Ashbrook, Anna
;
Koczwara, Katherine
;
Lek, Angela
, et al.
소스
Journal of Neuromuscular Diseases; September 2023, Vol. 10 Issue: 5 p797-812, 16p
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7 . Academic Journal
Visualizing Muscle Sialic Acid Expression in the GNE
D207V
Tg
Gne
-/-
Cmah
-/-
Model of GNE Myopathy: A Comparison of Dietary and Gene Therapy Approaches.
저자
by
Crowe, Kelly E.
;
Zygmunt, Deborah A.
;
Heller, Kristin
;
Rodino-Klapac, Louise
;
Noguchi, Satoru
, et al.
소스
Journal of Neuromuscular Diseases; 2022, Vol. 9 Issue 1, p53-71, 19p
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7 . Periodical
Visualizing Muscle Sialic Acid Expression in the GNED207VTgGne-/-Cmah-/-Model of GNE Myopathy: A Comparison of Dietary and Gene Therapy Approaches
저자
by
Crowe, Kelly E.
;
Zygmunt, Deborah A.
;
Heller, Kristin
;
Rodino-Klapac, Louise
;
Noguchi, Satoru
, et al.
소스
Journal of Neuromuscular Diseases; January 2022, Vol. 9 Issue: 1 p53-71, 19p
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7 . Academic Journal
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.
저자
by
Martin, Paul T.
;
Zygmunt, Deborah A.
;
Ashbrook, Anna
;
Hamilton, Sonia
;
Packer, Davin
, et al.
소스
PLoS ONE
. 3/26/2021, Vol. 16 Issue 3, p1-31. 31p.
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7 . Periodical
rAAVrh74.MCK.GALGT2Protects against Loss of Hemodynamic Function in the Aging mdxMouse Heart
저자
by
Xu, Rui
;
Jia, Ying
;
Zygmunt, Deborah A.
;
Martin, Paul T.
.
소스
Molecular Therapy; March 2019, Vol. 27 Issue: 3 p636-649, 14p
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