Results 251 to 260 of about 51,494 (318)

Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy. [PDF]

J Muscle Res Cell Motil
Chwalenia K, Wood MJA, Roberts TC.
europepmc   +1 more source

MET targeted therapy in non-small cell lung cancer patients with MET exon 14-skipping mutations

Jan Trøst Jørgensen, Edyta Maria Urbanska, Jens Mollerup   +2 more
openalex   +1 more source

Protocol for using MYOD1-transduced human urine-derived cells as a predictive platform for exon skipping therapy in Duchenne muscular dystrophy. [PDF]

STAR Protoc
Kunitake K, Ishizuka T, Takeshita E, Komaki H, Aoki Y.   +4 more
europepmc   +1 more source

Additional file 3: of Elusive sources of variability of dystrophin rescue by exon skipping

, 2015
Maria Candida Vila, Margaret Klimek, James S. Novak, Sree Rayavarapu, Kitipong Uaesoontrachoon, J Boehler, Alyson A. Fiorillo, Marshall W. Hogarth, Aiping Zhang, Conner J. Shaughnessy, Heather Gordish-Dressman, U. Burki, Volker Straub, Qi Lu, Terence Partridge, Kristy Brown, Yetrib Hathout, John N. van den Anker, Eric Hoffman, Kanneboyina Nagaraju   +19 more
openalex   +1 more source

Novel FBN1 intron variant causes isolated ectopia lentis via in-frame exon skipping. [PDF]

J Hum Genet
Shimizu N, Mashimo Y, Yokouchi H, Nishio Y, Sawai S, Ichikawa T, Ogi T, Baba T, Onouchi Y.   +8 more
europepmc   +1 more source

31 | Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy

Interuniversity Institute of Myology
openalex   +1 more source

30 Years Since the Proposal of Exon Skipping Therapy for Duchenne Muscular Dystrophy and the Future of Pseudoexon Skipping. [PDF]

Int J Mol Sci
Matsuo M.
europepmc   +1 more source

The silent mutation nucleotide 744 G → A, Lys172Lys, in exon 6 of BRCA2 results in exon skipping

, 2009
Thomas van Overeem Hansen, Ane Y. Steffensen, Lars Jønson, Mette Klarskov Andersen, Bent Ejlertsen, Finn C. Nielsen   +5 more
openalex   +2 more sources

Optimization of Exon-Skipping Riboswitches and Their Applications to Control Mammalian Cell Fate. [PDF]

ACS Synth Biol
Nomura Y, Kim N, Zhu B, Hamzah M, Zhang H, Yokobayashi Y.   +5 more
europepmc   +1 more source

Case Report: a novel variant in <i>WT1</i> leads to focal segmental glomerulosclerosis and uterovaginal anomalies through exon skipping. [PDF]

Front Nephrol
Marquez J, O'Sullivan L, Squire AE, Ryan GL, Debiec KE, Amies Oelschlager AM, Adam MP.   +6 more
europepmc   +1 more source