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The Journal of Clinical Investigation
|
September 4, 2019
Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
Arnaud F Klein, Miguel A Varela, Ludovic Arandel, et al.
Plos Genetics
|
December 5, 2012
Molecular, physiological, and motor performance defects in DMSXL mice carrying >1,000 CTG repeats from the human DM1 locus
Aline Huguet, Fadia Medja, Annie Nicole, et al.
The Journal of Clinical Investigation
|
July 16, 2004
Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle
Yvan Torrente, Marzia Belicchi, Maurilio Sampaolesi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 28, 2017
CRISPR/Cas9-Induced (CTG⋅CAG)<sub>n</sub> Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing
Ellen L van Agtmaal, Laurène M André, Marieke Willemse, et al.
Molecular Therapy. Nucleic Acids
|
November 4, 2015
In Vitro and In Vivo Modulation of Alternative Splicing by the Biguanide Metformin
Delphine Laustriat, Jacqueline Gide, Laetitia Barrault, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
August 12, 2009
Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy
Susan A M Mulders, Walther J A A van den Broek, Thurman M Wheeler, et al.
Nature Structural & Molecular Biology
|
June 21, 2011
Misregulation of miR-1 processing is associated with heart defects in myotonic dystrophy
Frédérique Rau, Fernande Freyermuth, Charlotte Fugier, et al.
Neuropathology and Applied Neurobiology
|
December 28, 2022
MBNL-dependent impaired development within the neuromuscular system in myotonic dystrophy type 1
Julie Tahraoui-Bories, Antoine Mérien, Anchel González-Barriga, et al.
JAMA Neurology
|
February 7, 2018
Development and Validation of a New Scoring System to Predict Survival in Patients With Myotonic Dystrophy Type 1
Karim Wahbi, Raphaël Porcher, Pascal Laforêt, et al.
Human Molecular Genetics
|
November 2, 2013
High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines
Ami Ketley, Catherine Z Chen, Xin Li, et al.
Page
of 8
Search research articles
Search
Showing results (61-70 of 80) with videos related to
Sort By:
Page
of 8
The Journal of Clinical Investigation
|
September 4, 2019
Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
Arnaud F Klein, Miguel A Varela, Ludovic Arandel, et al.
Plos Genetics
|
December 5, 2012
Molecular, physiological, and motor performance defects in DMSXL mice carrying >1,000 CTG repeats from the human DM1 locus
Aline Huguet, Fadia Medja, Annie Nicole, et al.
The Journal of Clinical Investigation
|
July 16, 2004
Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle
Yvan Torrente, Marzia Belicchi, Maurilio Sampaolesi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 28, 2017
CRISPR/Cas9-Induced (CTG⋅CAG)<sub>n</sub> Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing
Ellen L van Agtmaal, Laurène M André, Marieke Willemse, et al.
Molecular Therapy. Nucleic Acids
|
November 4, 2015
In Vitro and In Vivo Modulation of Alternative Splicing by the Biguanide Metformin
Delphine Laustriat, Jacqueline Gide, Laetitia Barrault, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
August 12, 2009
Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy
Susan A M Mulders, Walther J A A van den Broek, Thurman M Wheeler, et al.
Nature Structural & Molecular Biology
|
June 21, 2011
Misregulation of miR-1 processing is associated with heart defects in myotonic dystrophy
Frédérique Rau, Fernande Freyermuth, Charlotte Fugier, et al.
Neuropathology and Applied Neurobiology
|
December 28, 2022
MBNL-dependent impaired development within the neuromuscular system in myotonic dystrophy type 1
Julie Tahraoui-Bories, Antoine Mérien, Anchel González-Barriga, et al.
JAMA Neurology
|
February 7, 2018
Development and Validation of a New Scoring System to Predict Survival in Patients With Myotonic Dystrophy Type 1
Karim Wahbi, Raphaël Porcher, Pascal Laforêt, et al.
Human Molecular Genetics
|
November 2, 2013
High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines
Ami Ketley, Catherine Z Chen, Xin Li, et al.
Page
of 8