X
Search Filters
Format Format
Subjects Subjects
Subjects Subjects
X
Sort by Item Count (A-Z)
Filter by Count
index medicus (9) 9
mice (8) 8
animals (7) 7
humans (7) 7
medicine, research & experimental (6) 6
biochemistry & molecular biology (4) 4
exons (4) 4
expression (4) 4
life sciences (4) 4
alternative splicing (3) 3
cell biology (3) 3
dependovirus - genetics (3) 3
duchenne muscular-dystrophy (3) 3
genetic vectors (3) 3
genetics & heredity (3) 3
human health and pathology (3) 3
muscle, skeletal - metabolism (3) 3
mutations (3) 3
original (3) 3
original article (3) 3
physiological aspects (3) 3
research (3) 3
trans-splicing (3) 3
article (2) 2
binding-protein-c (2) 2
biotechnology & applied microbiology (2) 2
cell line (2) 2
cells (2) 2
cells, cultured (2) 2
centronuclear myopathy (2) 2
dilated cardiomyopathy (2) 2
dystrophin - genetics (2) 2
female (2) 2
gene (2) 2
gene transfer techniques (2) 2
gene-therapy (2) 2
gene-transfer (2) 2
genetic aspects (2) 2
genetic therapy (2) 2
genetic vectors - metabolism (2) 2
genetics (2) 2
health aspects (2) 2
hypertrophic cardiomyopathy (2) 2
introns (2) 2
mice, inbred c57bl (2) 2
mice, inbred mdx (2) 2
molecular biology (2) 2
muscle fibers, skeletal - metabolism (2) 2
muscular dystrophy, duchenne - genetics (2) 2
musculoskeletal diseases (2) 2
musculoskeletal system (2) 2
protein isoforms - genetics (2) 2
research articles (2) 2
risk factors (2) 2
rna (2) 2
rna splice sites (2) 2
rna, small nuclear - genetics (2) 2
skeletal-muscle (2) 2
transduction, genetic (2) 2
viruses (2) 2
3t3 cells (1) 1
[ sdv.mhep ] life sciences [q-bio]/human health and pathology (1) 1
[ sdv.mhep.csc ] life sciences [q-bio]/human health and pathology/cardiology and cardiovascular system (1) 1
acetylcholine-receptor clusters (1) 1
actin (1) 1
actin cytoskeleton - metabolism (1) 1
actinin - metabolism (1) 1
actins - metabolism (1) 1
adaptor proteins, signal transducing (1) 1
adaptor proteins, signal transducing - genetics (1) 1
adaptor proteins, signal transducing - physiology (1) 1
adenoassociated viral vectors (1) 1
adenoviridae - genetics (1) 1
aged (1) 1
aged, 80 and over (1) 1
alpha-actinin (1) 1
alternative splicing - physiology (1) 1
amphiphysin-2 bin1 (1) 1
analysis (1) 1
antigens, cd34 - metabolism (1) 1
antisense oligonucleotides (1) 1
antisense oligoribonucleotide (1) 1
binding proteins (1) 1
biosynthesis (1) 1
blood pressure (1) 1
blood pressure determination - methods (1) 1
cancer (1) 1
cardiac gene-transfer (1) 1
cardiac myocytes (1) 1
cardiac myosin- binding protein-c (1) 1
cardiac myosin-binding protein-c (1) 1
cardiac myosin‐binding protein‐c (1) 1
cardiology and cardiovascular system (1) 1
cardiomyopathy (1) 1
cardiomyopathy, hypertrophic - genetics (1) 1
cardiomyopathy, hypertrophic - pathology (1) 1
cardiomyopathy, hypertrophic - physiopathology (1) 1
cardiomyopathy, hypertrophic - therapy (1) 1
cardiotoxins - pharmacology (1) 1
cardiovascular disease (1) 1
more...
Language Language
Publication Date Publication Date
Click on a bar to filter by decade
Slide to change publication date range


Nature Medicine, ISSN 1078-8956, 2011, Volume 17, Issue 6, pp. 720 - 725
Myotonic dystrophy is the most common muscular dystrophy in adults and the first recognized example of an RNA-mediated disease. Congenital myotonic dystrophy... 
MEDICINE, RESEARCH & EXPERIMENTAL | CTG REPEAT | CENTRONUCLEAR MYOPATHY | PROTEIN-KINASE | BIOCHEMISTRY & MOLECULAR BIOLOGY | MYOTUBULAR MYOPATHY | CELL BIOLOGY | SKELETAL-MUSCLE | MESSENGER-RNA | GENE | CHLORIDE CHANNEL | AMPHIPHYSIN-2 BIN1 | MUTATIONS | Cell Line | RNA-Binding Proteins - physiology | Humans | Alternative Splicing - physiology | Exons - genetics | Muscle Weakness - genetics | Protein Isoforms - physiology | Tumor Suppressor Proteins - physiology | Adaptor Proteins, Signal Transducing - physiology | Myotonic Dystrophy - physiopathology | Animals | Tumor Suppressor Proteins - genetics | Adaptor Proteins, Signal Transducing - genetics | Myotonic Dystrophy - genetics | Mice | Nuclear Proteins - physiology | Muscle Weakness - physiopathology | Nuclear Proteins - genetics | Muscle Fibers, Skeletal - physiology | RNA-Binding Proteins - metabolism | Protein Isoforms - genetics | Muscle weakness | Myotonic dystrophy | RNA | Physiological aspects | Genetic aspects | Research | Risk factors | Musculoskeletal diseases | Musculoskeletal system | Biosynthesis | Protein synthesis | Muscular dystrophy | Index Medicus | Alternative Splicing | Exons | Myotonic Dystrophy | RNA-Binding Proteins | Nuclear Proteins | Life Sciences | Adaptor Proteins, Signal Transducing | Muscle Fibers, Skeletal | Protein Isoforms | Muscle Weakness | Tumor Suppressor Proteins | Cancer | Naturvetenskap | Natural Sciences
Journal Article
Journal of Cell Biology, ISSN 0021-9525, 2014, Volume 205, Issue 3, pp. 377 - 393
Journal Article
EMBO Molecular Medicine, ISSN 1757-4676, 07/2013, Volume 5, Issue 7, pp. 1128 - 1145
Exon skipping is a promising therapy for selected genetic diseases. Here, the authors show as a proof‐of‐principle that MYBPC3 mutation‐induced cardiomyopathy... 
cardiac myosin‐binding protein‐C | hypertrophic cardiomyopathy | alternative splicing | exon skipping | antisense oligoribonucleotide | Hypertrophic cardiomyopathy | Alternative splicing | Antisense oligoribonucleotide | Cardiac myosin-binding protein-C | Exon skipping | MEDICINE, RESEARCH & EXPERIMENTAL | BINDING-PROTEIN-C | RESTORES DYSTROPHIN EXPRESSION | UBIQUITIN-PROTEASOME SYSTEM | cardiac myosin-binding protein-C | SPLICE DONOR SITE | DILATED CARDIOMYOPATHY | CARDIAC GENE-TRANSFER | DUCHENNE MUSCULAR-DYSTROPHY | MUTATIONS | ANTISENSE OLIGONUCLEOTIDES | Genetic Therapy | Alternative Splicing | Exons | Humans | Oligoribonucleotides, Antisense - administration & dosage | RNA, Small Nuclear - administration & dosage | RNA, Small Nuclear - genetics | Myocardium - metabolism | HEK293 Cells | Adenoviridae - genetics | Cardiomyopathy, Hypertrophic - therapy | Heart - physiopathology | RNA, Small Nuclear - therapeutic use | Cardiomyopathy, Hypertrophic - genetics | Oligoribonucleotides, Antisense - therapeutic use | Transduction, Genetic | Hypertrophy, Left Ventricular - prevention & control | RNA, Messenger - genetics | Myocardium - pathology | Gene Knock-In Techniques | Carrier Proteins - genetics | Animals | Cardiomyopathy, Hypertrophic - physiopathology | Oligoribonucleotides, Antisense - genetics | Mice | Mutation | Cardiomyopathy, Hypertrophic - pathology | Protein Isoforms - genetics | Cardiomyopathy | Health aspects | Heart diseases | Analysis | Medical research | Rodents | Index Medicus | Life Sciences | Human health and pathology | Cardiology and cardiovascular system
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 2013, Volume 2, Issue 7, pp. e102 - e102
RNA -splicing has been explored as a therapeutic option for a variety of genetic diseases, but not for cardiac genetic disease. Hypertrophic cardiomyopathy... 
hypertrophic cardiomyopathy | trans-splicing | Mybpc3 | RNA-based therapy | MEDICINE, RESEARCH & EXPERIMENTAL | BINDING-PROTEIN-C | DIAGNOSIS | SENSITIVITY | DILATED CARDIOMYOPATHY | ADENOASSOCIATED VIRAL VECTORS | DISEASE | GENE-THERAPY | MICE | MUTATIONS | EXPRESSION | Life Sciences | Human health and pathology | Original
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 2015, Volume 2, pp. 15024 - 15024
Recombinant adeno-associated viruses (rAAV) are promising candidates for gene therapy approaches. The last two decades were particularly fruitful in terms of... 
MEDICINE, RESEARCH & EXPERIMENTAL
Journal Article
by Hannedouche, Thierry and Roth, Hubert and Krummel, Thierry and London, Gérard M and Jean, Guillaume and Bouchet, Jean-Louis and Drüeke, Tilman B and Fouque, Denis and Attroun, Lahcene and Azar, Raymond and Bories, Pierre and Caillette-Beaudouin, Agnès and Canaud, Bernard and Choukroun, Gabriel and Esnault, Vincent and Hammadi, Mohamed and Henri, Patrick and Honoré, Philippe and Issad, Belkacem and Joly, Dominique and Laruelle, Eric and Le Mao, Gildas and Marchais, Sylvain and Vendrely, Benoît and Zaoui, Philippe and Aazib, Larbi and Abbassi, Abdelhamid and Abdullah, Elias and Abou-Bekr, Habib and Achard-Hottelart, Carine and Achin, Geneviève and Ahriz-Saksi, Salima and Albadawy, Mahen and Albert, Catherine and Albitar, Samir and Alenabi, Farideh and Allouache, Mahmoud and Amaouche, Amar and Amara, Brahim and Ammor, Mounia and Ang, Kim Seng and Assogba, Ubald and Azzouz, Lynda and Badid, Chérif and Baleynaud, Juliette and Bargas, Evelyne and Baron, Emmanuel and Basse, François and Batho, Jean-Marie and Bauwens, Marc and Bazin, Dorothée and Aicha, Abdelmajid Ben and Benarbia, Seddik and Bencheikh, Larbi and Bendini, Jean-Christophe and Benyakoub, Djeleddine and Bergua, Dominique and Bessin, Catherine and Billaux, Luc and Billion, Stéphane and Bittar, Haïat and Bocquet, Jean-Paul and Bonarek, Hervé and Bonniol, Claude and Borde, Jean-Sébastien and Boubenider, Samir and Boudet, Rémi and Boudi, Waël and Boudier, Loreley and Bouguern, Djema and Boukelmoune, M and Boukhalfa, Fatha Zohra and Boulanger, Henri and Bouvier, Philippe and Bouzernidj, Mouloud and Bounab, Mohamed Brahim and Brasseur, José and Braun, Laura and Briet, Marie and Bui-Quang, Doan and Canet, Sebastien and Canivet, Eric and Cardozo, Carlos and Cardozo, Jorge and Chaghouri, Baher and Chawki, Mokhtar and Chazot, Charles and Choulet, Philippe and Clavel, Pierre and Coindre, Jean-Philippe and Coldefy, Olivier and Colomina, M.A and Combarnous, François and Dabot, Christian and Dahmane, Djamal and Dahmani, Ahmed and Daubresse, Daniel and De Fremont, Jean-François and De Precigout, Valérie and Dehais, Françoise and ... and French Observ and French Observatory
Kidney International, ISSN 0085-2538, 09/2016, Volume 90, Issue 3, pp. 674 - 684
Journal Article
Molecular Therapy: Methods & Clinical Development, ISSN 2329-0501, Volume 2
Recombinant adeno-associated viruses (rAAV) are promising candidates for gene therapy approaches. The last two decades were particularly fruitful in terms of... 
Journal Article