Zobrazeno 1 - 10
of 33
pro vyhledávání: '"Liubov V Gushchina"'
Autor:
Liubov V. Gushchina, Adrienne J. Bradley, Tatyana A. Vetter, Jacob W. Lay, Natalie L. Rohan, Emma C. Frair, Nicolas Wein, Kevin M. Flanigan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101144- (2023)
Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by mutations in the DMD gene that prevent the expression of a functional dystrophin protein. Exon duplications represent 6%–11% of mutations, and duplications of exon 2 (Dup
Externí odkaz:
https://doaj.org/article/1dee77955bc4458f84bf563a278aee74
Autor:
Liubov V. Gushchina, Tatyana A. Vetter, Emma C. Frair, Adrienne J. Bradley, Kelly M. Grounds, Jacob W. Lay, Nianyuan Huang, Aisha Suhaiba, Frederick J. Schnell, Gunnar Hanson, Tabatha R. Simmons, Nicolas Wein, Kevin M. Flanigan
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 30, Iss , Pp 479-492 (2022)
Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease that arises due to the loss of dystrophin expression, leading to progressive loss of motor and cardiorespiratory function. Four exon-skipping approaches using antisense phospho
Externí odkaz:
https://doaj.org/article/c58f1dc6613c474f8ea734b55a626d47
Autor:
Nicolas Wein, Tatyana A. Vetter, Adeline Vulin, Tabatha R. Simmons, Emma C. Frair, Adrienne J. Bradley, Liubov V. Gushchina, Camila F. Almeida, Nianyuan Huang, Daniel Lesman, Dhanarajan Rajakumar, Robert B. Weiss, Kevin M. Flanigan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 279-293 (2022)
Duchenne muscular dystrophy (DMD) is typically caused by mutations that disrupt the DMD reading frame, but nonsense mutations in the 5′ part of the gene induce utilization of an internal ribosomal entry site (IRES) in exon 5, driving expression of
Externí odkaz:
https://doaj.org/article/937ffb28cf15459caaa9793b8cebec7b
Autor:
Shuting Zhao, Zhaobin Xu, Hai Wang, Benjamin E. Reese, Liubov V. Gushchina, Meng Jiang, Pranay Agarwal, Jiangsheng Xu, Mingjun Zhang, Rulong Shen, Zhenguo Liu, Noah Weisleder, Xiaoming He
Publikováno v:
Nature Communications, Vol 7, Iss 1, Pp 1-12 (2016)
Stem cell therapy of myocardial infarction is hampered by poor survival of injected cells. Here the authors develop injectable aggregates of stem cells differentiated to an early cardiac stage and encapsulated in a biodegradable micromatrix, and show
Externí odkaz:
https://doaj.org/article/366e57a1bda54b80a00b5fe9ce11e6aa
Autor:
Michelle Eggers, Nicolas Wein, Hemantkumar D Chavan, Kevin M. Flanigan, Adrienne J Bradley, Liubov V Gushchina, Emma C Frair, Hsin-Jung Chou, Megan A. Waldrop, Tabatha R. Simmons, Natalie Rohan
Publikováno v:
Human Gene Therapy. 32:882-894
Therapeutic exon skipping as a treatment for Duchenne muscular dystrophy (DMD) has largely concentrated on the delivery of antisense oligomers to treat out-of-frame exon deletions. Here we report o...
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::5e6a19be41010c71837d9b049f88e3ad
https://doi.org/10.1089/hum.2020.286
https://doi.org/10.1089/hum.2020.286
Autor:
Diane M. Dunn, Robert B. Weiss, Megan A. Waldrop, Nicolas Wein, Emma C Frair, Kevin M. Flanigan, Liubov V Gushchina
Publikováno v:
Human Gene Therapy.
Exon skipping therapies for Duchenne muscular dystrophy that restore an open reading frame can be induced by the use of non-coding U7 small nuclear RNA (U7snRNA) modified by an antisense exon-targeting sequence delivered by an adeno-associated virus
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::d9ed0eeb9fb795c5605d09bd17f05f1b
https://doi.org/10.1089/hum.2020.315
https://doi.org/10.1089/hum.2020.315
Autor:
Nicolas, Wein, Diane M, Dunn, Megan A, Waldrop, Liubov V, Gushchina, Emma C, Frair, Robert B, Weiss, Kevin M, Flanigan
Publikováno v:
Human gene therapy. 32(21-22)
Exon skipping therapies for Duchenne muscular dystrophy that restore an open reading frame can be induced by the use of noncoding U7 small nuclear RNA (U7snRNA) modified by an antisense exon-targeting sequence delivered by an adeno-associated virus (
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::d729ce8ecef49001653a5610ea1c3941
https://pubmed.ncbi.nlm.nih.gov/34060935
https://pubmed.ncbi.nlm.nih.gov/34060935
Autor:
Liubov V, Gushchina, Emma C, Frair, Natalie, Rohan, Adrienne J, Bradley, Tabatha R, Simmons, Hemantkumar D, Chavan, Hsin-Jung, Chou, Michelle, Eggers, Megan A, Waldrop, Nicolas, Wein, Kevin M, Flanigan
Publikováno v:
Human gene therapy. 32(17-18)
Therapeutic exon skipping as a treatment for Duchenne muscular dystrophy (DMD) has largely concentrated on the delivery of antisense oligomers to treat out-of-frame exon deletions. Here we report on the preclinical development of an adeno-associated
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::95fe550ec7788f1301027bef02b71ee5
https://pubmed.ncbi.nlm.nih.gov/33406986
https://pubmed.ncbi.nlm.nih.gov/33406986
Publikováno v:
Pharmacology & Therapeutics. 185:12-25
The tripartite motif (TRIM) gene family is a highly conserved group of E3 ubiquitin ligase proteins that can establish substrate specificity for the ubiquitin-proteasome complex and also have proteasome-independent functions. While several family mem
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3867b21ebf8244d660d95d561b2665fc
https://doi.org/10.1016/j.pharmthera.2017.10.020
https://doi.org/10.1016/j.pharmthera.2017.10.020
Autor:
Jin Hyuk Choi, Heather R. Manring, Noah Weisleder, Sayak Bhattacharya, Jenna Alloush, Liubov V. Gushchina, Kevin E. McElhanon, Eric X Beck
Publikováno v:
Molecular Therapy. 25:2360-2371
Limb girdle muscular dystrophy type 2B (LGMD2B) and other dysferlinopathies are degenerative muscle diseases that result from mutations in the dysferlin gene and have limited treatment options. The dysferlin protein has been linked to multiple cellul
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::70f7a6b5f4b940165ddd381d56a1f460
https://doi.org/10.1016/j.ymthe.2017.06.025
https://doi.org/10.1016/j.ymthe.2017.06.025