Zobrazeno 1 - 10
of 30
pro vyhledávání: '"Joel R. Chamberlain"'
Autor:
Niclas E. Bengtsson, John K. Hall, Guy L. Odom, Michael P. Phelps, Colin R. Andrus, R. David Hawkins, Stephen D. Hauschka, Joel R. Chamberlain, Jeffrey S. Chamberlain
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-10 (2017)
CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrop
Externí odkaz:
https://doaj.org/article/42b84f4d2d7e485cb3c0f8f3e7eaf353
Autor:
Niclas E. Bengtsson, John K. Hall, Guy L. Odom, Michael P. Phelps, Colin R. Andrus, R. David Hawkins, Stephen D. Hauschka, Joel R. Chamberlain, Jeffrey S. Chamberlain
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-1 (2017)
Nature Communications 8: Article number: 14454 (2017); Published: 14 February 2017; Updated: 23 June 2017 This Article contains an error in Fig. 4, for which we apologize. In panel a, the image reporting dystrophin labelling following SaCas9Δ5253 tr
Externí odkaz:
https://doaj.org/article/77235027c66448b889ec5dc2c4163e56
Publikováno v:
Molecular Therapy. Nucleic Acids
Gene knockdown using short hairpin RNAs (shRNAs) is a promising strategy for targeting dominant mutations; however, delivering too much shRNA can disrupt the processing of endogenous microRNAs (miRNAs) and lead to toxicity. Here, we sought to underst
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ec62a66c7124acf7d3b9e21156c1aa49
https://doi.org/10.1016/j.omtn.2019.12.007
https://doi.org/10.1016/j.omtn.2019.12.007
Autor:
Andrea LH Arnett, Patryk Konieczny, Julian N Ramos, John Hall, Guy Odom, Zipora Yablonka-Reuveni, Joel R Chamberlain, Jeffrey S Chamberlain
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Adeno-associated viral (AAV) vectors are becoming an important tool for gene therapy of numerous genetic and other disorders. Several recombinant AAV vectors (rAAV) have the ability to transduce striated muscles in a variety of animals following intr
Externí odkaz:
https://doaj.org/article/6dcdb3beee8246b0bdbf94909a612092
Autor:
Jessica Wei, Joel R Chamberlain
Publikováno v:
PLoS ONE, Vol 9, Iss 8, p e102053 (2014)
RNAi has potential for therapeutically downregulating the expression of dominantly inherited genes in a variety of human genetic disorders. Here we used the ROSA26 mouse, which constitutively expresses the bacterial lacZ gene in tissues body wide, as
Externí odkaz:
https://doaj.org/article/7c6f85cee7a441dd80cf71635c82f6a2
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 25(5)
Duchenne muscular dystrophy (DMD) has been a major target for gene therapy development for nearly 30 years. DMD is among the most common genetic diseases, and isolation of the defective gene (DMD, or dystrophin) was a landmark discovery, as it was th
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f7cc6b356872553f16d99449ca3e2504
https://pubmed.ncbi.nlm.nih.gov/28416280
https://pubmed.ncbi.nlm.nih.gov/28416280
Autor:
Michael Phelps, Joel R. Chamberlain, John K. Hall, Jeffrey S. Chamberlain, Colin Andrus, Niclas E. Bengtsson, Guy L. Odom, R. David Hawkins, Stephen D. Hauschka
Publikováno v:
Nature Communications
Nature Communications, Vol 8, Iss 1, Pp 1-10 (2017)
Nature Communications, Vol 8, Iss 1, Pp 1-10 (2017)
Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors to edit specific regions of the DMD gene using CRISPR/Cas9. Here we devel
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::feaf5c3201b50c06c0611204ab12ca66
http://europepmc.org/articles/PMC5316861
http://europepmc.org/articles/PMC5316861
Autor:
Joel R. Chamberlain, David R. Deyle, David W. Russell, Roli K. Hirata, Pei-Rong Wang, Peter H. Byers, Ulrike Schwarze, Yi Li
Publikováno v:
Molecular Therapy. 16:187-193
Mesenchymal stem cells (MSCs) are adult cells with the capacity to differentiate into multiple cell types, including bone, fat, cartilage, and muscle cells. In order to effectively utilize autologous MSCs in cell-based therapies, precise genetic mani
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b4acb57f50e0f4e41555803d8574267c
https://doi.org/10.1038/sj.mt.6300339
https://doi.org/10.1038/sj.mt.6300339
Autor:
Ewa Stepniak-Konieczna, Gregory T. Carter, Darren R. Bisset, Joel R. Chamberlain, Maja Zavaljevski, Jessica Wei, Michael D. Weiss
RNA interference (RNAi) offers a promising therapeutic approach for dominant genetic disorders that involve gain-of-function mechanisms. One candidate disease for RNAi therapy application is myotonic dystrophy type 1 (DM1), which results from toxicit
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a7e69efd97e7b548b67a2efd0bd36d26
https://europepmc.org/articles/PMC4527493/
https://europepmc.org/articles/PMC4527493/
Publikováno v:
Nature Biotechnology. 20:735-738
Efficient methods are needed for the precise genetic manipulation of diploid human cells, in which cellular senescence and low conventional gene targeting rates limit experimental and therapeutic options. We have shown previously that linear, single-
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::05a6c9536f22fc7b1e058c5947bb1958
https://doi.org/10.1038/nbt0702-735
https://doi.org/10.1038/nbt0702-735