Stephen D Hauschka
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Explore the profile of Stephen D Hauschka including associated specialties, affiliations and a list of published articles.
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46
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1627
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Recent Articles
1.
Tasfaout H, Halbert C, McMillen T, Allen J, Reyes T, Flint G, et al.
Nature
. 2024 Jul;
632(8023):192-200.
PMID: 39020181
Gene replacement using adeno-associated virus (AAV) vectors is a promising therapeutic approach for many diseases. However, this therapeutic modality is challenged by the packaging capacity of AAVs (approximately 4.7 kilobases),...
2.
Karbassi E, Padgett R, Bertero A, Reinecke H, Klaiman J, Yang X, et al.
Cell Mol Life Sci
. 2024 Feb;
81(1):95.
PMID: 38372898
Human induced pluripotent stem cells (hiPSCs) offer opportunities to study human biology where primary cell types are limited. CRISPR technology allows forward genetic screens using engineered Cas9-expressing cells. Here, we...
3.
Mhatre K, Mathieu J, Martinson A, Flint G, Blakley L, Tabesh A, et al.
bioRxiv
. 2023 May;
PMID: 37162854
Transplanted human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs) improve ventricular performance when delivered acutely post-myocardial infarction but are ineffective in chronic myocardial infarction/heart failure. 2'-deoxy-ATP (dATP) activates cardiac myosin and potently...
4.
Birch S, Lawlor M, Conlon T, Guo L, Crudele J, Hawkins E, et al.
Sci Transl Med
. 2023 Jan;
15(677):eabo1815.
PMID: 36599002
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystrophin, a membrane-stabilizing protein encoded by the gene. Although mouse models of DMD provide insight...
5.
Bengtsson N, Crudele J, Klaiman J, Halbert C, Hauschka S, Chamberlain J
Mol Ther
. 2022 Feb;
30(6):2176-2185.
PMID: 35143959
Gene editing has shown promise for correcting or bypassing dystrophin mutations in Duchenne muscular dystrophy (DMD). However, preclinical studies have focused on young animals with limited muscle fibrosis and wasting,...
6.
Garg K, Mahmassani Z, Dvoretskiy S, Valero M, Huntsman H, Lapp S, et al.
J Gerontol A Biol Sci Med Sci
. 2020 Dec;
76(4):586-590.
PMID: 33284954
Anabolic resistance to a mechanical stimulus may contribute to the loss of skeletal muscle mass observed with age. In this study, young and aged mice were injected with saline or...
7.
Bengtsson N, Tasfaout H, Hauschka S, Chamberlain J
Mol Ther
. 2020 Nov;
29(3):1070-1085.
PMID: 33160075
Gene editing is often touted as a permanent method for correcting mutations, but its long-term benefits in Duchenne muscular dystrophy (DMD) may depend on sufficiently high editing efficiencies to halt...
8.
Kwon J, Ettyreddy A, Vankara A, Bohning J, Devlin G, Hauschka S, et al.
Mol Ther Methods Clin Dev
. 2020 Nov;
19:320-329.
PMID: 33145368
Delivery of therapeutic transgenes with adeno-associated viral (AAV) vectors for treatment of myopathies has yielded encouraging results in animal models and early clinical studies. Although certain AAV serotypes efficiently target...
9.
Kolwicz Jr S, Hall J, Moussavi-Harami F, Chen X, Hauschka S, Chamberlain J, et al.
JACC Basic Transl Sci
. 2020 Jan;
4(7):778-791.
PMID: 31998848
Mutations in the gene encoding for dystrophin leads to structural and functional deterioration of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients. Administration of recombinant...
10.
Ramos J, Hollinger K, Bengtsson N, Allen J, Hauschka S, Chamberlain J
Mol Ther
. 2019 Feb;
27(3):623-635.
PMID: 30718090
Gene therapies using adeno-associated viral (AAV) vectors have advanced into clinical trials for several diseases, including Duchenne muscular dystrophy (DMD). A limitation of AAV is the carrying capacity (∼5 kb)...