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pro vyhledávání: '"Rebecca C. Schray"'
Autor:
Rebecca C. Schray, Jason H. Williams, Michelle L. Erney, Shashank R. Sirsi, Nicole M. Lykens, Xiangying Guan, Gordon J. Lutz
Publikováno v:
Human Gene Therapy. 19:795-806
Exon-skipping oligonucleotides (ESOs) with 2'-O-methyl modifications are promising compounds for the treatment of Duchenne muscular dystrophy (DMD). However, the usefulness of these compounds is limited by their poor delivery profile to muscle tissue
Publikováno v:
Brain research. 1408
Aldehyde dehydrogenase 1A1 (ALDH1A1) is a member of a superfamily of detoxification enzymes found in various tissues that participate in the oxidation of both aliphatic and aromatic aldehydes. In the brain, ALDH1A1 participates in the metabolism of c
Autor:
Rebecca C. Schray, Jason H. Williams, Carlyn A. Patterson, Melanie K. Tallent, Gordon J. Lutz, Semira O. Ayitey
Spinal muscular atrophy (SMA) is caused by homozygous mutation or deletion of the SMN1 gene encoding survival of motor neuron (SMN) protein, resulting in the selective loss of alpha-motor neurons. Humans typically have one or more copies of the SMN2
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::41ef1a83d058c9e17c7f5345e13a79b9
https://europepmc.org/articles/PMC6665627/
https://europepmc.org/articles/PMC6665627/
Publikováno v:
Journal of Nanobiotechnology
Journal of Nanobiotechnology, Vol 7, Iss 1, p 1 (2009)
Journal of Nanobiotechnology, Vol 7, Iss 1, p 1 (2009)
Antisense oligonucleotides (AOs) have been shown to induce dystrophin expression in muscles cells of patients with Duchenne Muscular Dystrophy (DMD) and in the mdx mouse, the murine model of DMD. However, ineffective delivery of AOs limits their ther
Autor:
Shashank R. Sirsi, Rebecca C. Schray, Xiangying Guan, Nicole M. Lykens, Jason H. Williams, Michelle L. Erney, Gordon J. Lutz
Publikováno v:
Human Gene Therapy; Aug2008, Vol. 19 Issue 8, p795-806, 12p
Publikováno v:
BMC Biotechnology
BMC Biotechnology, Vol 8, Iss 1, p 35 (2008)
BMC Biotechnology, Vol 8, Iss 1, p 35 (2008)
Background Exon skipping oligonucleotides (ESOs) of 2'O-Methyl (2'OMe) and morpholino chemistry have been shown to restore dystrophin expression in muscle fibers from the mdx mouse, and are currently being tested in phase I clinical trials for Duchen